Program Publications
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The search results on this publication page are automated on a monthly schedule based on acknowledgement of NIH Common Fund award numbers and intramural awards. Therefore, this list is not an exhaustive or error-free account of the program’s publications.
Title | Authors | Journal | PubMedID | Publication Date |
---|---|---|---|---|
Production of High-Titer Retrovirus and Lentivirus Vectors. | Sena-Esteves, Miguel; Gao, Guangping | Cold Spring Harbor protocols. | 29610362 | 2018 04 02; |
Titration of Lentivirus Vectors. | Sena-Esteves, Miguel; Gao, Guangping | Cold Spring Harbor protocols. | 29610363 | 2018 04 02; |
Monitoring Lentivirus Vector Stocks for Replication-Competent Viruses. | Sena-Esteves, Miguel; Gao, Guangping | Cold Spring Harbor protocols. | 29610364 | 2018 04 02; |
H3Africa: current perspectives. | Mulder, Nicola; Abimiku, Alash'le; Adebamowo, Sally N; de Vries, Jantina; Matimba, Alice; Olowoyo, Paul; Ramsay, Michele; Skelton, Michelle; Stein, Dan J | Pharmacogenomics and personalized medicine. | 29692621 | 2018; |
Clinical Evaluations of Toxicity and Efficacy of Nanoparticle-Mediated Gene Therapy. | Liang, Xiao; Liu, Li; Wei, Yu-Quan; Gao, Guang-Ping; Wei, Xia-Wei | Human gene therapy. | 29893153 | 2018 11; |
Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice. | Wang, Dan; Li, Jia; Song, Chun-Qing; Tran, Karen; Mou, Haiwei; Wu, Pei-Hsuan; Tai, Phillip W L; Mendonca, Craig A; Ren, Lingzhi; Wang, Blake Y; Su, Qin; Gessler, Dominic J; Zamore, Phillip D; Xue, Wen; Gao, Guangping | Nature biotechnology. | 30102296 | 2018 Oct; |
Tunability of Biodegradable Poly(amine- co-ester) Polymers for Customized Nucleic Acid Delivery and Other Biomedical Applications. | Kauffman, Amy C; Piotrowski-Daspit, Alexandra S; Nakazawa, Kay H; Jiang, Yuhang; Datye, Amit; Saltzman, W Mark | Biomacromolecules | 30110158 | 2018 09 10 |
The temporal landscape of recursive splicing during Pol II transcription elongation in human cells. | Zhang, Xiao-Ou; Fu, Yu; Mou, Haiwei; Xue, Wen; Weng, Zhiping | PLoS genetics. | 30148885 | 2018 08; |
All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo. | Ibraheim, Raed; Song, Chun-Qing; Mir, Aamir; Amrani, Nadia; Xue, Wen; Sontheimer, Erik J | Genome biology. | 30231914 | 2018 Sep 19; |
Defining CRISPR-Cas9 genome-wide nuclease activities with CIRCLE-seq. | Lazzarotto, Cicera R; Nguyen, Nhu T; Tang, Xing; Malagon-Lopez, Jose; Guo, Jimmy A; Aryee, Martin J; Joung, J Keith; Tsai, Shengdar Q | Nature protocols. | 30341435 | 2018 11; |
Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy. | Gong, Yi; Berenson, Anna; Laheji, Fiza; Gao, Guangping; Wang, Dan; Ng, Carrie; Volak, Adrienn; Kok, Rene; Kreouzis, Vasileios; Dijkstra, Inge M; Kemp, Stephan; Maguire, Casey A; Eichler, Florian | Human gene therapy. | 30358470 | 2019 May; |
Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer. | Wang, Dan; Zhong, Li; Li, Mengxin; Li, Jia; Tran, Karen; Ren, Lingzhi; He, Ran; Xie, Jun; Moser, Richard P; Fraser, Cara; Kuchel, Tim; Sena-Esteves, Miguel; Flotte, Terence R; Aronin, Neil; Gao, Guangping | Molecular therapy. Methods & clinical development. | 30397628 | 2018 Dec 14; |
Understanding and repurposing CRISPR-mediated alternative splicing. | Smith, Jordan L; Mou, Haiwei; Xue, Wen | Genome biology. | 30400804 | 2018 11 06; |
MicroRNA-96 Promotes Schistosomiasis Hepatic Fibrosis in Mice by Suppressing Smad7. | Luo, Xufeng; Zhang, Dongmei; Xie, Jun; Su, Qin; He, Xing; Bai, Ruipu; Gao, Guangping; Pan, Weiqing | Molecular therapy. Methods & clinical development. | 30406154 | 2018 Dec 14; |
Targeting fidelity of adenine and cytosine base editors in mouse embryos. | Lee, Hye Kyung; Willi, Michaela; Miller, Shannon M; Kim, Sojung; Liu, Chengyu; Liu, David R; Hennighausen, Lothar | Nature communications. | 30442934 | 2018 Nov 15; |
Intracellular Delivery of His-Tagged Genome-Editing Proteins Enabled by Nitrilotriacetic Acid-Containing Lipidoid Nanoparticles. | Li, Yamin; Li, Alice Chukun; Xu, Qiaobing | Advanced healthcare materials. | 30565897 | 2019 Mar; |
Illuminating the genome-wide activity of genome editors for safe and effective therapeutics. | Cheng, Yong; Tsai, Shengdar Q | Genome biology. | 30577870 | 2018 12 22; |
A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing. | Edraki, Alireza; Mir, Aamir; Ibraheim, Raed; Gainetdinov, Ildar; Yoon, Yeonsoo; Song, Chun-Qing; Cao, Yueying; Gallant, Judith; Xue, Wen; Rivera-Pérez, Jaime A; Sontheimer, Erik J | Molecular cell. | 30581144 | 2018 Dec 18; |
Integrating Combinatorial Lipid Nanoparticle and Chemically Modified Protein for Intracellular Delivery and Genome Editing. | Chang, Jin; Chen, Xianghan; Glass, Zachary; Gao, Feng; Mao, Lanqun; Wang, Ming; Xu, Qiaobing | Accounts of chemical research. | 30586281 | 2019 Mar 19; |
Enhancing the In Vitro and In Vivo Stabilities of Polymeric Nucleic Acid Delivery Nanosystems. | Wang, Yuyuan; Ye, Mingzhou; Xie, Ruosen; Gong, Shaoqin | Bioconjugate chemistry. | 30592619 | 2019 02 20; |
Anti-drug Antibody Responses Impair Prophylaxis Mediated by AAV-Delivered HIV-1 Broadly Neutralizing Antibodies. | Gardner, Matthew R; Fetzer, Ina; Kattenhorn, Lisa M; Davis-Gardner, Meredith E; Zhou, Amber S; Alfant, Barnett; Weber, Jesse A; Kondur, Hema R; Martinez-Navio, Jose M; Fuchs, Sebastian P; Desrosiers, Ronald C; Gao, Guangping; Lifson, Jeffrey D; Farzan, Mi | Molecular therapy : the journal of the American Society of Gene Therapy. | 30704961 | 2019 03 06; |
Adeno-associated virus vector as a platform for gene therapy delivery. | Wang, Dan; Tai, Phillip W L; Gao, Guangping | Nature reviews. Drug discovery. | 30710128 | 2019 05; |
Simultaneous targeting of linked loci in mouse embryos using base editing. | Lee, Hye Kyung; Willi, Michaela; Smith, Harold E; Miller, Shannon M; Liu, David R; Liu, Chengyu; Hennighausen, Lothar | Scientific reports. | 30733567 | 2019 Feb 07; |
Quantitative and Digital Droplet-Based AAV Genome Titration. | Sanmiguel, Julio; Gao, Guangping; Vandenberghe, Luk H | Methods in molecular biology (Clifton, N.J.). | 30783968 | 2019; |
Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System. | Gessler, Dominic J; Tai, Phillip W L; Li, Jia; Gao, Guangping | Methods in molecular biology (Clifton, N.J.). | 30783972 | 2019; |
Kidney organoids in translational medicine: Disease modeling and regenerative medicine. | Miyoshi, Tomoya; Hiratsuka, Ken; Saiz, Edgar Garcia; Morizane, Ryuji | Developmental dynamics : an official publication of the American Association of Anatomists | 30843293 | 2020 01 |
Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression. | Martinez-Navio, José M; Fuchs, Sebastian P; Pantry, Shara N; Lauer, William A; Duggan, Natasha N; Keele, Brandon F; Rakasz, Eva G; Gao, Guangping; Lifson, Jeffrey D; Desrosiers, Ronald C | Immunity. | 30850342 | 2019 Mar 19; |
Enhanced Cas12a editing in mammalian cells and zebrafish. | Liu, Pengpeng; Luk, Kevin; Shin, Masahiro; Idrizi, Feston; Kwok, Samantha; Roscoe, Benjamin; Mintzer, Esther; Suresh, Sneha; Morrison, Kyle; Frazão, Josias B; Bolukbasi, Mehmet Fatih; Ponnienselvan, Karthikeyan; Luban, Jeremy; Zhu, Lihua Julie; Lawson, Nathan D; Wolfe, Scot A | Nucleic acids research. | 30892626 | 2019 Mar 20; |
An orange calcium-modulated bioluminescent indicator for non-invasive activity imaging. | Oh, Younghee; Park, Yunhee; Cho, Julia H; Wu, Haodi; Paulk, Nicole K; Liu, Lan Xiang; Kim, Namdoo; Kay, Mark A; Wu, Joseph C; Lin, Michael Z | Nature chemical biology. | 30936501 | 2019 05; |
CRISPR-SONIC: targeted somatic oncogene knock-in enables rapid in vivo cancer modeling. | Mou, Haiwei; Ozata, Deniz M; Smith, Jordan L; Sheel, Ankur; Kwan, Suet-Yan; Hough, Soren; Kucukural, Alper; Kennedy, Zachary; Cao, Yueying; Xue, Wen | Genome medicine. | 30987660 | 2019 04 16; |
Unbiased detection of CRISPR off-targets in vivo using DISCOVER-Seq. | Wienert, Beeke; Wyman, Stacia K; Richardson, Christopher D; Yeh, Charles D; Akcakaya, Pinar; Porritt, Michelle J; Morlock, Michaela; Vu, Jonathan T; Kazane, Katelynn R; Watry, Hannah L; Judge, Luke M; Conklin, Bruce R; Maresca, Marcello; Corn, Jacob E | Science (New York, N.Y.). | 31000663 | 2019 04 19; |
Nucleic Acid Therapeutics for Neurological Diseases. | Watts, Jonathan K; Brown, Robert H; Khvorova, Anastasia | Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics. | 31037650 | 2019 04; |
Construction of Recombinant Adenovirus Genomes by Direct Cloning. | Zhou, Xiangyang; Sena-Esteves, Miguel; Gao, Guangping | Cold Spring Harbor protocols. | 31043558 | 2019 05 01; |
Release of the Cloned Recombinant Adenovirus Genome for Rescue and Expansion. | Su, Qin; Sena-Esteves, Miguel; Gao, Guangping | Cold Spring Harbor protocols. | 31043559 | 2019 05 01; |
Purification of the Recombinant Adenovirus by Cesium Chloride Gradient Centrifugation. | Su, Qin; Sena-Esteves, Miguel; Gao, Guangping | Cold Spring Harbor protocols. | 31043560 | 2019 05 01; |
High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs. | Xu, Meiyu; Li, Jia; Xie, Jun; He, Ran; Su, Qin; Gao, Guangping; Tai, Phillip W L | Human gene therapy. | 31072208 | 2019 Jun 17; |
Analysis and minimization of cellular RNA editing by DNA adenine base editors. | Rees, Holly A; Wilson, Christopher; Doman, Jordan L; Liu, David R | Science advances. | 31086823 | 2019 May; |
Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks. | Rees, Holly A; Yeh, Wei-Hsi; Liu, David R | Nature communications. | 31101808 | 2019 05 17; |
Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors. | Huang, Tony P; Zhao, Kevin T; Miller, Shannon M; Gaudelli, Nicole M; Oakes, Benjamin L; Fellmann, Christof; Savage, David F; Liu, David R | Nature biotechnology. | 31110355 | 2019 Jun; |
Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity. | Xiao, Yuanyuan; Muhuri, Manish; Li, Shaoyong; Qin, Wanru; Xu, Guangchao; Luo, Li; Li, Jia; Letizia, Alexander J; Wang, Sean K; Chan, Ying Kai; Wang, Chunmei; Fuchs, Sebastian P; Wang, Dan; Su, Qin; Nahid, M Abu; Church, George M; Farzan, Michael; Yang, Li; Wei, Yuquan; Desrosiers, Ronald C; Mueller, Christian; Tai, Phillip Wl; Gao, Guangping | JCI insight. | 31112525 | 2019 May 21; |
Depletion of TRRAP Induces p53-Independent Senescence in Liver Cancer by Down-Regulating Mitotic Genes. | Kwan, Suet-Yan; Sheel, Ankur; Song, Chun-Qing; Zhang, Xiao-Ou; Jiang, Tingting; Dang, Hien; Cao, Yueying; Ozata, Deniz M; Mou, Haiwei; Yin, Hao; Weng, Zhiping; Wang, Xin Wei; Xue, Wen | Hepatology (Baltimore, Md.). | 31188495 | 2019 Jun 12; |
Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles. | Liu, Ji; Chang, Jin; Jiang, Ying; Meng, Xiandi; Sun, Tianmeng; Mao, Lanqun; Xu, Qiaobing; Wang, Ming | Advanced materials (Deerfield Beach, Fla.). | 31215123 | 2019 Jun 19; |
An anionic human protein mediates cationic liposome delivery of genome editing proteins into mammalian cells. | Kim, Y Bill; Zhao, Kevin T; Thompson, David B; Liu, David R | Nature communications. | 31266953 | 2019 07 02; |
Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis. | Yang, Yeon-Suk; Xie, Jun; Wang, Dan; Kim, Jung-Min; Tai, Phillip W L; Gravallese, Ellen; Gao, Guangping; Shim, Jae-Hyuck | Nature communications. | 31273195 | 2019 07 04; |
Continuous evolution of base editors with expanded target compatibility and improved activity. | Thuronyi, Benjamin W; Koblan, Luke W; Levy, Jonathan M; Yeh, Wei-Hsi; Zheng, Christine; Newby, Gregory A; Wilson, Christopher; Bhaumik, Mantu; Shubina-Oleinik, Olga; Holt, Jeffrey R; Liu, David R | Nature biotechnology. | 31332326 | 2019 Jul 22; |
Nonviral Nanoparticles for CRISPR-Based Genome Editing: Is It Just a Simple Adaption of What Have Been Developed for Nucleic Acid Delivery? | Qiu, Min; Glass, Zachary; Xu, Qiaobing | Biomacromolecules. | 31342740 | 2019 09 09; |
Mapping cis-regulatory chromatin contacts in neural cells links neuropsychiatric disorder risk variants to target genes. | Song, Michael; Yang, Xiaoyu; Ren, Xingjie; Maliskova, Lenka; Li, Bingkun; Jones, Ian R; Wang, Chao; Jacob, Fadi; Wu, Kenneth; Traglia, Michela; Tam, Tsz Wai; Jamieson, Kirsty; Lu, Si-Yao; Ming, Guo-Li; Li, Yun; Yao, Jun; Weiss, Lauren A; Dixon, Jesse R; J | Nature genetics. | 31367015 | 2019 08; |
Measuring the Infectious Titer of Recombinant Adenovirus Using Tissue Culture Infection Dose 50% (TCID50) End-Point Dilution and Quantitative Polymerase Chain Reaction (qPCR). | Lock, Martin; Korn, Michael; Wilson, James; Sena-Esteves, Miguel; Gao, Guangping | Cold Spring Harbor protocols. | 31371467 | 2019 Aug 01; |
Preparation of a DNA Standard for Quantitative Polymerase Chain Reaction (qPCR). | Lock, Martin; Korn, Michael; Wilson, James; Sena-Esteves, Miguel; Gao, Guangping | Cold Spring Harbor protocols. | 31371468 | 2019 Aug 01; |
Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs. | Osborn, Mark J; Newby, Gregory A; McElroy, Amber N; Knipping, Friederike; Nielsen, Sarah C; Riddle, Megan J; Xia, Lily; Chen, Weili; Eide, Cindy R; Webber, Beau R; Wandall, Hans H; Dabelsteen, Sally; Blazar, Bruce R; Liu, David R; Tolar, Jakub | The Journal of investigative dermatology. | 31437443 | 2020 Feb; |
Tissue-restricted genome editing in vivo specified by microRNA-repressible anti-CRISPR proteins. | Lee, Jooyoung; Mou, Haiwei; Ibraheim, Raed; Liang, Shun-Qing; Liu, Pengpeng; Xue, Wen; Sontheimer, Erik J | RNA (New York, N.Y.). | 31439808 | 2019 11; |
Noninvasive imaging of renal urea handling by CEST-MRI. | Shin, Soo Hyun; Wendland, Michael F; Zhang, Brandon; Tran, An; Tang, Albert; Vandsburger, Moriel H | Magnetic resonance in medicine | 31483529 | 2020 03 |
A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing. | Chen, Guojun; Abdeen, Amr A; Wang, Yuyuan; Shahi, Pawan K; Robertson, Samantha; Xie, Ruosen; Suzuki, Masatoshi; Pattnaik, Bikash R; Saha, Krishanu; Gong, Shaoqin | Nature nanotechnology. | 31501532 | 2019 Oct; |
Blueprint for building a biorepository in a resource-limited setting that follows international best practices. | Abimiku, Alash'le G; Croxton, Talishea; Ozumba, Petronilla J; Agala, Ndidi; Balogun, Olasinbo; Jonathan, Emmanuel; Onyemata, Enzenwa; Ndifon, Kachimi; Nadoma, Sunji; Anazodo, Thankgod; Peters, Sam; Beiswanger, Christine M | African journal of laboratory medicine. | 31534913 | 2019; |
CRISPR genome editing in stem cells turns to gold. | Asokan, Aravind | Nature materials | 31537943 | 2019 10 |
Search-and-replace genome editing without double-strand breaks or donor DNA. | Anzalone, Andrew V; Randolph, Peyton B; Davis, Jessie R; Sousa, Alexander A; Koblan, Luke W; Levy, Jonathan M; Chen, Peter J; Wilson, Christopher; Newby, Gregory A; Raguram, Aditya; Liu, David R | Nature. | 31634902 | 2019 12; |
Optimizing biodegradable nanoparticle size for tissue-specific delivery. | Mandl, Hanna K; Quijano, Elias; Suh, Hee Won; Sparago, Emily; Oeck, Sebastian; Grun, Molly; Glazer, Peter M; Saltzman, W Mark | Journal of controlled release : official journal of the Controlled Release Society. | 31654688 | 2019 Nov 28; |
Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia. | Krishnamurthy, Sateesh; Wohlford-Lenane, Christine; Kandimalla, Suhas; Sartre, Gilles; Meyerholz, David K; Théberge, Vanessa; Hallée, Stéphanie; Duperré, Anne-Marie; Del'Guidice, Thomas; Lepetit-Stoffaes, Jean-Pascal; Barbeau, Xavier; Guay, David; McCray | Nature communications. | 31659165 | 2019 10 28; |
Structures of Neisseria meningitidis Cas9 Complexes in Catalytically Poised and Anti-CRISPR-Inhibited States. | Sun, Wei; Yang, Jing; Cheng, Zhi; Amrani, Nadia; Liu, Chao; Wang, Kangkang; Ibraheim, Raed; Edraki, Alireza; Huang, Xue; Wang, Min; Wang, Jiuyu; Liu, Liang; Sheng, Gang; Yang, Yanhua; Lou, Jizhong; Sontheimer, Erik J; Wang, Yanli | Molecular cell. | 31668930 | 2019 12 19; |
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. | Métais, Jean-Yves; Doerfler, Phillip A; Mayuranathan, Thiyagaraj; Bauer, Daniel E; Fowler, Stephanie C; Hsieh, Matthew M; Katta, Varun; Keriwala, Sagar; Lazzarotto, Cicera R; Luk, Kevin; Neel, Michael D; Perry, S Scott; Peters, Samuel T; Porter, Shaina N; | Blood advances. | 31698466 | 2019 11 12; |
Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids. | Huang, Qin; Chan, Ken Y; Tobey, Isabelle G; Chan, Yujia Alina; Poterba, Tim; Boutros, Christine L; Balazs, Alejandro B; Daneman, Richard; Bloom, Jonathan M; Seed, Cotton; Deverman, Benjamin E | PloS one. | 31725765 | 2019; |
Gene Delivery to Nonhuman Primate Preimplantation Embryos Using Recombinant Adeno-Associated Virus. | Wang, Dan; Niu, Yuyu; Ren, Lingzhi; Kang, Yu; Tai, Phillip W L; Si, Chenyang; Mendonca, Craig A; Ma, Hong; Gao, Guangping; Ji, Weizhi | Advanced science (Weinheim, Baden-Wurttemberg, Germany). | 31728271 | 2019 Nov 06; |
Adenine base editing in an adult mouse model of tyrosinaemia. | Song, Chun-Qing; Jiang, Tingting; Richter, Michelle; Rhym, Luke H; Koblan, Luke W; Zafra, Maria Paz; Schatoff, Emma M; Doman, Jordan L; Cao, Yueying; Dow, Lukas E; Zhu, Lihua Julie; Anderson, Daniel G; Liu, David R; Yin, Hao; Xue, Wen | Nature biomedical engineering. | 31740768 | 2020 Jan; |
Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings. | Maynard, Lucy H; Smith, Olivia; Tilmans, Nicolas P; Tham, Eleonore; Hosseinzadeh, Shayan; Tan, Weilun; Leenay, Ryan; May, Andrew P; Paulk, Nicole K | Human gene therapy methods. | 31855083 | 2019 12; |
Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses. | Levy, Jonathan M; Yeh, Wei-Hsi; Pendse, Nachiket; Davis, Jessie R; Hennessey, Erin; Butcher, Rossano; Koblan, Luke W; Comander, Jason; Liu, Qin; Liu, David R | Nature biomedical engineering. | 31937940 | 2020 Jan; |
Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications. | Li, Yamin; Glass, Zachary; Huang, Mingqian; Chen, Zheng-Yi; Xu, Qiaobing | Biomaterials. | 31945616 | 2020 Mar; |
Detection of Extracellular Vesicle RNA Using Molecular Beacons. | Oliveira Jr, Getulio Pereira de; Zigon, Eric; Rogers, Gaenna; Davodian, Danny; Lu, Shulin; Jovanovic-Talisman, Tijana; Jones, Jennifer; Tigges, John; Tyagi, Sanjay; Ghiran, Ionita Calin | iScience. | 31958756 | 2020 Jan 24; |
From GWAS Association to Function: Candidate Gene Screening Within Insulin Resistance-Associated Genomic Loci Using a Preadipocyte Differentiation Model. | Ladha, Feria A; Stitzel, Michael L; Hinson, J Travis | Circulation research. | 31999535 | 2020 Jan 31; |
Quantitating Endosomal Escape of a Library of Polymers for mRNA Delivery. | Jiang, Yuhang; Lu, Qiao; Wang, Yongheng; Xu, Emily; Ho, Alison; Singh, Priya; Wang, Yifei; Jiang, Zhaozhong; Yang, Fan; Tietjen, Gregory T; Cresswell, Peter; Saltzman, W Mark | Nano letters. | 32003222 | 2020 Feb 12; |
High-throughput quantitative microscopy-based half-life measurements of intravenously injected agents. | Bracaglia, Laura G; Piotrowski-Daspit, Alexandra S; Lin, Chun-Yu; Moscato, Zoe M; Wang, Yongheng; Tietjen, Gregory T; Saltzman, W Mark | Proceedings of the National Academy of Sciences of the United States of America. | 32015123 | 2020 Feb 18; |
Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors. | Doman, Jordan L; Raguram, Aditya; Newby, Gregory A; Liu, David R | Nature biotechnology. | 32042165 | 2020 Feb 10; |
Continuous evolution of SpCas9 variants compatible with non-G PAMs. | Miller, Shannon M; Wang, Tina; Randolph, Peyton B; Arbab, Mandana; Shen, Max W; Huang, Tony P; Matuszek, Zaneta; Newby, Gregory A; Rees, Holly A; Liu, David R | Nature biotechnology. | 32042170 | 2020 Apr; |
Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair. | Economos, Nicholas G; Oyaghire, Stanley; Quijano, Elias; Ricciardi, Adele S; Saltzman, W Mark; Glazer, Peter M | Molecules (Basel, Switzerland). | 32046275 | 2020 Feb 08; |
Clades of huge phages from across Earth's ecosystems. | Al-Shayeb, Basem; Sachdeva, Rohan; Chen, Lin-Xing; Ward, Fred; Munk, Patrick; Devoto, Audra; Castelle, Cindy J; Olm, Matthew R; Bouma-Gregson, Keith; Amano, Yuki; He, Christine; Méheust, Raphaël; Brooks, Brandon; Thomas, Alex; Lavy, Adi; Matheus-Carnevali | Nature. | 32051592 | 2020 02; |
The promise and challenge of therapeutic genome editing. | Doudna, Jennifer A | Nature | 32051598 | 2020 02 |
Site-Specific Post-translational Surface Modification of Adeno-Associated Virus Vectors Using Leucine Zippers. | Thadani, Nicole N; Yang, Joanna; Moyo, Buhle; Lee, Ciaran M; Chen, Maria Y; Bao, Gang; Suh, Junghae | ACS synthetic biology. | 32068391 | 2020 Mar 20; |
Poly(Lactic-co-Glycolic Acid) Nanoparticle Delivery of Peptide Nucleic Acids In Vivo. | Oyaghire, Stanley N; Quijano, Elias; Piotrowski-Daspit, Alexandra S; Saltzman, W Mark; Glazer, Peter M | Methods in molecular biology (Clifton, N.J.). | 32088877 | 2020; |
A New Gorilla Adenoviral Vector with Natural Lung Tropism Avoids Liver Toxicity and Is Amenable to Capsid Engineering and Vector Retargeting. | Lu, Zhi Hong; Dmitriev, Igor P; Brough, Douglas E; Kashentseva, Elena A; Li, Jie; Curiel, David T | Journal of virology | 32102889 | 2020 05 04 |
Can we mimic skeletal muscles for novel drug discovery? | Broer, Torie; Khodabukus, Alastair; Bursac, Nenad | Expert opinion on drug discovery. | 32122176 | 2020 Mar 03; |
Targeted Delivery of Secretory Promelittin via Novel Poly(lactone-co-?-amino ester) Nanoparticles for Treatment of Breast Cancer Brain Metastases. | Zhou, Yu; Zhang, Shenqi; Chen, Zeming; Bao, Youmei; Chen, Ann T; Sheu, Wendy C; Liu, Fuyao; Jiang, Zhaozhong; Zhou, Jiangbing | Advanced science (Weinheim, Baden-Wurttemberg, Germany). | 32154067 | 2020 Mar; |
CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors. | Wang, Dan; Zhang, Feng; Gao, Guangping | Cell. | 32243786 | 2020 Apr 02; |
Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases. | Wu, Shao-Shuai; Li, Qing-Cui; Yin, Chang-Qing; Xue, Wen; Song, Chun-Qing | Theranostics. | 32292501 | 2020; |
Tissue-Engineered Human Myobundle System as a Platform for Evaluation of Skeletal Muscle Injury Biomarkers. | Khodabukus, Alastair; Kaza, Amulya; Wang, Jason; Prabhu, Neel; Goldstein, Richard; Vaidya, Vishal S; Bursac, Nenad | Toxicological sciences : an official journal of the Society of Toxicology. | 32294208 | 2020 Jul 01; |
Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells. | Nagpal, Neha; Wang, Jianing; Zeng, Jing; Lo, Emily; Moon, Diane H; Luk, Kevin; Braun, Roman O; Burroughs, Lauri M; Keel, Sioban B; Reilly, Christopher; Lindsley, R Coleman; Wolfe, Scot A; Tai, Albert K; Cahan, Patrick; Bauer, Daniel E; Fong, Yick W; Agarwal, Suneet | Cell stem cell. | 32320679 | 2020 Jun 04; |
Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators. | Kwon, Jennifer B; Vankara, Ashish; Ettyreddy, Adarsh R; Bohning, Joel D; Gersbach, Charles A | Stem cell reports. | 32330446 | 2020 05 12; |
Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope. | Jiang, Tingting; Henderson, Jordana M; Coote, Kevin; Cheng, Yi; Valley, Hillary C; Zhang, Xiao-Ou; Wang, Qin; Rhym, Luke H; Cao, Yueying; Newby, Gregory A; Bihler, Hermann; Mense, Martin; Weng, Zhiping; Anderson, Daniel G; McCaffrey, Anton P; Liu, David R; Xue, Wen | Nature communications. | 32332735 | 2020 Apr 24; |
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9. | Li, Ang; Tanner, Mark R; Lee, Ciaran M; Hurley, Ayrea E; De Giorgi, Marco; Jarrett, Kelsey E; Davis, Timothy H; Doerfler, Alexandria M; Bao, Gang; Beeton, Christine; Lagor, William R | Molecular therapy : the journal of the American Society of Gene Therapy. | 32348718 | 2020 Jun 03; |
Positron emission tomography imaging of novel AAV capsids maps rapid brain accumulation. | Seo, Jai Woong; Ingham, Elizabeth S; Mahakian, Lisa; Tumbale, Spencer; Wu, Bo; Aghevlian, Sadaf; Shams, Shahin; Baikoghli, Mo; Jain, Poorva; Ding, Xiaozhe; Goeden, Nick; Dobreva, Tatyana; Flytzanis, Nicholas C; Chavez, Michael; Singhal, Kratika; Leib, Rya | Nature communications. | 32355221 | 2020 04 30; |
Finding order in slow axonal transport. | Roy, Subhojit | Current opinion in neurobiology. | 32361600 | 2020 08; |
Once upon a dish: engineering multicellular systems. | Haase, Kristina; Freedman, Benjamin S | Development (Cambridge, England). | 32366376 | 2020 May 04; |
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Prime genome editing in rice and wheat. | Lin, Qiupeng; Zong, Yuan; Xue, Chenxiao; Wang, Shengxing; Jin, Shuai; Zhu, Zixu; Wang, Yanpeng; Anzalone, Andrew V; Raguram, Aditya; Doman, Jordan L; Liu, David R; Gao, Caixia | Nature biotechnology. | 32393904 | 2020 05; |
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Protein and mRNA Delivery Enabled by Cholesteryl-Based Biodegradable Lipidoid Nanoparticles. | Li, Yamin; Jarvis, Rachel; Zhu, Kuixin; Glass, Zachary; Ogurlu, Roza; Gao, Peiyang; Li, Peixuan; Chen, Jinjin; Yu, Yingjie; Yang, Yongjie; Xu, Qiaobing | Angewandte Chemie (International ed. in English). | 32438474 | 2020 May 21; |
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Combinatorial Library of Cyclic Benzylidene Acetal-Containing pH-Responsive Lipidoid Nanoparticles for Intracellular mRNA Delivery. | Li, Yamin; Li, Raissa; Chakraborty, Anirban; Ogurlu, Roza; Zhao, Xuewei; Chen, Jinjin; Xu, Qiaobing | Bioconjugate chemistry. | 32520527 | 2020 07 15; |
Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy. | Li, Jixue; Li, Yanjie; Wang, Jun; Gonzalez, Trevor J; Asokan, Aravind; Napierala, Jill S; Napierala, Marek | Human gene therapy | 32527155 | 2020 08 |
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Induced pluripotent stem cells provide mega insights into kidney disease. | Luciani, Alessandro; Freedman, Benjamin S | Kidney international | 32571490 | 2020 07 |
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Programmable m6A modification of cellular RNAs with a Cas13-directed methyltransferase. | Wilson, Christopher; Chen, Peter J; Miao, Zhuang; Liu, David R | Nature biotechnology. | 32601430 | 2020 Jun 29; |
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Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties. | Hsu, Hung-Lun; Brown, Alexander; Loveland, Anna B; Lotun, Anoushka; Xu, Meiyu; Luo, Li; Xu, Guangchao; Li, Jia; Ren, Lingzhi; Su, Qin; Gessler, Dominic J; Wei, Yuquan; Tai, Phillip W L; Korostelev, Andrei A; Gao, Guangping | Nature communications. | 32606306 | 2020 Jun 30; |
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing. | Mok, Beverly Y; de Moraes, Marcos H; Zeng, Jun; Bosch, Dustin E; Kotrys, Anna V; Raguram, Aditya; Hsu, FoSheng; Radey, Matthew C; Peterson, S Brook; Mootha, Vamsi K; Mougous, Joseph D; Liu, David R | Nature. | 32641830 | 2020 07; |
A scoutRNA Is Required for Some Type V CRISPR-Cas Systems. | Harrington, Lucas B; Ma, Enbo; Chen, Janice S; Witte, Isaac P; Gertz, Dov; Paez-Espino, David; Al-Shayeb, Basem; Kyrpides, Nikos C; Burstein, David; Banfield, Jillian F; Doudna, Jennifer A | Molecular cell. | 32645367 | 2020 08 06; |
Profiling APOL1 Nephropathy Risk Variants in Genome-Edited Kidney Organoids with Single-Cell Transcriptomics. | Liu, Esther; Radmanesh, Behram; Chung, Byungha H; Donnan, Michael D; Yi, Dan; Dadi, Amal; Smith, Kelly D; Himmelfarb, Jonathan; Li, Mingyao; Freedman, Benjamin S; Lin, Jennie | Kidney360. | 32656538 | 2020 Mar; |
Imidazole-Based Synthetic Lipidoids for In Vivo mRNA Delivery into Primary T Lymphocytes. | Zhao, Xuewei; Chen, Jinjin; Qiu, Min; Li, Yamin; Glass, Zachary; Xu, Qiaobing | Angewandte Chemie (International ed. in English) | 32662132 | 2020 11 02 |
Advances in Alpha-1 Antitrypsin Gene Therapy. | Lorincz, Reka; Curiel, David T | American journal of respiratory cell and molecular biology. | 32668173 | 2020 Nov; |
Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach. | Havlik, L Patrick; Simon, Katherine E; Smith, J Kennon; Klinc, Kelli A; Tse, Longping V; Oh, Daniel K; Fanous, Marco M; Meganck, Rita M; Mietzsch, Mario; Kleinschmidt, Jürgen; Agbandje-McKenna, Mavis; Asokan, Aravind | Journal of virology. | 32669336 | 2020 Sep 15; |
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The dawn of non-human primate models for neurodevelopmental disorders. | Aida, Tomomi; Feng, Guoping | Current opinion in genetics & development | 32693220 | 2020 12 |
Advances in microphysiological blood-brain barrier (BBB) models towards drug delivery. | Lee, Caleb S; Leong, Kam W | Current opinion in biotechnology. | 32711361 | 2020 Dec; |
DNA capture by a CRISPR-Cas9-guided adenine base editor. | Lapinaite, Audrone; Knott, Gavin J; Palumbo, Cody M; Lin-Shiao, Enrique; Richter, Michelle F; Zhao, Kevin T; Beal, Peter A; Liu, David R; Doudna, Jennifer A | Science (New York, N.Y.). | 32732424 | 2020 07 31; |
Epigenetic transcriptional reprogramming by WT1 mediates a repair response during podocyte injury. | Ettou, Sandrine; Jung, Youngsook L; Miyoshi, Tomoya; Jain, Dhawal; Hiratsuka, Ken; Schumacher, Valerie; Taglienti, Mary E; Morizane, Ryuji; Park, Peter J; Kreidberg, Jordan A | Science advances. | 32754639 | 2020 Jul; |
NDAT Targets PI3K-Mediated PD-L1 Upregulation to Reduce Proliferation in Gefitinib-Resistant Colorectal Cancer. | Huang, Tung-Yung; Chang, Tung-Cheng; Chin, Yu-Tang; Pan, Yi-Shin; Chang, Wong-Jin; Liu, Feng-Cheng; Hastuti, Ema Dwi; Chiu, Shih-Jiuan; Wang, Shwu-Huey; Changou, Chun A; Li, Zi-Lin; Chen, Yi-Ru; Chu, Hung-Ru; Shih, Ya-Jung; Cheng, R Holland; Wu, Alexander | Cells. | 32756527 | 2020 08 03; |
Treating Cystic Fibrosis with mRNA and CRISPR. | Da Silva Sanchez, Alejandro; Paunovska, Kalina; Cristian, Ana; Dahlman, James E | Human gene therapy | 32799680 | 2020 09 |
New Directions in Pulmonary Gene Therapy. | Vu, Amber; McCray , Jr, Paul B | Human gene therapy | 32814451 | 2020 09 |
mRNA Delivery Using Bioreducible Lipidoid Nanoparticles Facilitates Neural Differentiation of Human Mesenchymal Stem Cells. | Zhao, Xuewei; Glass, Zachary; Chen, Jinjin; Yang, Liu; Kaplan, David L; Xu, Qiaobing | Advanced healthcare materials | 32815325 | 2021 02 |
The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction. | Madigan, Victoria J; Berry, Garrett E; Tyson, Tyne O; Nardone-White, Dasean; Ark, Jonathan; Elmore, Zachary C; Murlidharan, Giridhar; Vincent, Heather A; Asokan, Aravind | Journal of virology. | 32817219 | 2020 Oct 14; |
Opportunities and limitations of genetically modified nonhuman primate models for neuroscience research. | Feng, Guoping; Jensen, Frances E; Greely, Henry T; Okano, Hideyuki; Treue, Stefan; Roberts, Angela C; Fox, James G; Caddick, Sarah; Poo, Mu-Ming; Newsome, William T; Morrison, John H | Proceedings of the National Academy of Sciences of the United States of America. | 32817435 | 2020 09 29; |
Single C-to-T substitution using engineered APOBEC3G-nCas9 base editors with minimum genome- and transcriptome-wide off-target effects. | Lee, Sangsin; Ding, Ning; Sun, Yidi; Yuan, Tanglong; Li, Jing; Yuan, Qichen; Liu, Lizhong; Yang, Jie; Wang, Qian; Kolomeisky, Anatoly B; Hilton, Isaac B; Zuo, Erwei; Gao, Xue | Science advances | 32832622 | 2020 07 |
Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection. | Ma, Feihe; Yang, Liu; Sun, Zhuorui; Chen, Jinjin; Rui, Xuehui; Glass, Zachary; Xu, Qiaobing | Science advances | 32832671 | 2020 07 |
Frame-Hydrogel Methodology for Engineering Highly Functional Cardiac Tissue Constructs. | Helfer, Abbigail; Bursac, Nenad | Methods in molecular biology (Clifton, N.J.). | 32857373 | 2021; |
Synthetic immunomodulation with a CRISPR super-repressor in vivo. | Moghadam, Farzaneh; LeGraw, Ryan; Velazquez, Jeremy J; Yeo, Nan Cher; Xu, Chenxi; Park, Jin; Chavez, Alejandro; Ebrahimkhani, Mo R; Kiani, Samira | Nature cell biology. | 32884147 | 2020 Sep; |
Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors. | Boucher, Paul; Cui, Xiaoxia; Curiel, David T | Journal of controlled release : official journal of the Controlled Release Society | 32891680 | 2020 11 10 |
Rapid, precise quantification of large DNA excisions and inversions by ddPCR. | Watry, Hannah L; Feliciano, Carissa M; Gjoni, Ketrin; Takahashi, Gou; Miyaoka, Yuichiro; Conklin, Bruce R; Judge, Luke M | Scientific reports. | 32913194 | 2020 09 10; |
SARS-CoV-2 infection of human iPSC-derived cardiac cells predicts novel cytopathic features in hearts of COVID-19 patients. | Pérez-Bermejo, Juan A; Kang, Serah; Rockwood, Sarah J; Simoneau, Camille R; Joy, David A; Ramadoss, Gokul N; Silva, Ana C; Flanigan, Will R; Li, Huihui; Nakamura, Ken; Whitman, Jeffrey D; Ott, Melanie; Conklin, Bruce R; McDevitt, Todd C | bioRxiv : the preprint server for biology. | 32935097 | 2020 Sep 12; |
Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme. | Elmore, Zachary C; Oh, Daniel K; Simon, Katherine E; Fanous, Marco M; Asokan, Aravind | JCI insight | 32941184 | 2020 09 17 |
Serum Concentrations of Per- and Polyfluoroalkyl Substances and Risk of Renal Cell Carcinoma. | Shearer, Joseph J; Callahan, Catherine L; Calafat, Antonia M; Huang, Wen-Yi; Jones, Rena R; Sabbisetti, Venkata S; Freedman, Neal D; Sampson, Joshua N; Silverman, Debra T; Purdue, Mark P; Hofmann, Jonathan N | Journal of the National Cancer Institute | 32944748 | 2021 May 04 |
Direct Functional Protein Delivery with a Peptide into Neonatal and Adult Mammalian Inner Ear In Vivo. | Zhang, Kun; Cheng, Xiaoting; Zhao, Liping; Huang, Mingqian; Tao, Yong; Zhang, Hongbo; Rosenholm, Jessica M; Zhuang, Min; Chen, Zheng-Yi; Chen, Bing; Shu, Yilai | Molecular therapy. Methods & clinical development. | 32953927 | 2020 Sep 11; |
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors. | Rumachik, Neil G; Malaker, Stacy A; Poweleit, Nicole; Maynard, Lucy H; Adams, Christopher M; Leib, Ryan D; Cirolia, Giana; Thomas, Dennis; Stamnes, Susan; Holt, Kathleen; Sinn, Patrick; May, Andrew P; Paulk, Nicole K | Molecular therapy. Methods & clinical development. | 32995354 | 2020 Sep 11; |
COVID-19 CG: Tracking SARS-CoV-2 mutations by locations and dates of interest. | Chen, Albert Tian; Altschuler, Kevin; Zhan, Shing Hei; Chan, Yujia Alina; Deverman, Benjamin E | bioRxiv : the preprint server for biology. | 32995794 | 2020 Sep 28; |
Development of a Cardiac Sarcomere Functional Genomics Platform to Enable Scalable Interrogation of Human TNNT2 Variants. | Pettinato, Anthony M; Ladha, Feria A; Mellert, David J; Legere, Nicholas; Cohn, Rachel; Romano, Robert; Thakar, Ketan; Chen, Yu-Sheng; Hinson, J Travis | Circulation. | 33025817 | 2020 Oct 07; |
3D kidney organoids for bench-to-bedside translation. | Gupta, Navin; Dilmen, Emre; Morizane, Ryuji | Journal of molecular medicine (Berlin, Germany). | 33034708 | 2020 Oct 09; |
A Versatile Nonviral Delivery System for Multiplex Gene-Editing in the Liver. | Gong, Jing; Wang, Hong-Xia; Lao, Yeh-Hsing; Hu, Hanze; Vatan, Naazanene; Guo, Jonathan; Ho, Tzu-Chieh; Huang, Dantong; Li, Mingqiang; Shao, Dan; Leong, Kam W | Advanced materials (Deerfield Beach, Fla.) | 33053221 | 2020 Nov |
Reactions to the National Academies/Royal Society Report on Heritable Human Genome Editing. | Angrist, Misha; Barrangou, Rodolphe; Baylis, Françoise; Brokowski, Carolyn; Burgio, Gaetan; Caplan, Arthur; Chapman, Carolyn Riley; Church, George M; Cook-Deegan, Robert; Cwik, Bryan; Doudna, Jennifer A; Evans, John H; Greely, Henry T; Hercher, Laura; Hurlbut, J Benjamin; Hynes, Richard O; Ishii, Tetsuya; Kiani, Samira; Lee, LaTasha Hoskins; Levrier, Guillaume; Liu, David R; Lunshof, Jeantine E; Macintosh, Kerry Lynn; Mathews, Debra J H; Meslin, Eric M; Mills, Peter H R; Montoliu, Lluis; Musunuru, Kiran; Nicol, Dianne; O'Neill, Helen; Qiu, Renzong; Ranisch, Robert; Sherkow, Jacob S; Soni, Sheetal; Terry, Sharon; Topol, Eric; Williamson, Robert; Zhang, Feng; Davies, Kevin | The CRISPR journal | 33095048 | 2020 10 |
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy. | Kwon, Jennifer B; Ettyreddy, Adarsh R; Vankara, Ashish; Bohning, Joel D; Devlin, Garth; Hauschka, Stephen D; Asokan, Aravind; Gersbach, Charles A | Molecular therapy. Methods & clinical development. | 33145368 | 2020 Dec 11; |
Poison Exon Splicing Regulates a Coordinated Network of SR Protein Expression during Differentiation and Tumorigenesis. | Leclair, Nathan K; Brugiolo, Mattia; Urbanski, Laura; Lawson, Shane C; Thakar, Ketan; Yurieva, Marina; George, Joshy; Hinson, John Travis; Cheng, Albert; Graveley, Brenton R; Anczuków, Olga | Molecular cell. | 33176162 | 2020 11 19; |
Redirecting Vesicular Transport to Improve Nonviral Delivery of Molecular Cargo. | Mao, Mao; Chang, Chun-Chi; Pickar-Oliver, Adrian; Cervia, Lisa D; Wang, Liangli; Ji, Jing; Liton, Paloma B; Gersbach, Charles A; Yuan, Fan | Advanced biosystems | 33179869 | 2020 08 |
Delayed urea differential enhancement CEST (dudeCEST)-MRI with T1 correction for monitoring renal urea handling. | Shin, Soo Hyun; Wendland, Michael F; Vandsburger, Moriel H | Magnetic resonance in medicine. | 33180343 | 2020 Nov 12; |
The once and future gene therapy. | Bulaklak, Karen; Gersbach, Charles A | Nature communications. | 33199717 | 2020 11 16; |
Phage-assisted continuous and non-continuous evolution. | Miller, Shannon M; Wang, Tina; Liu, David R | Nature protocols. | 33199872 | 2020 Dec; |
Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation Screens. | Black, Joshua B; McCutcheon, Sean R; Dube, Shataakshi; Barrera, Alejandro; Klann, Tyler S; Rice, Grayson A; Adkar, Shaunak S; Soderling, Scott H; Reddy, Timothy E; Gersbach, Charles A | Cell reports. | 33264623 | 2020 Dec 01; |
Tools for experimental and computational analyses of off-target editing by programmable nucleases. | Bao, X Robert; Pan, Yidan; Lee, Ciaran M; Davis, Timothy H; Bao, Gang | Nature protocols. | 33288953 | 2020 Dec 07; |
Gene Regulatory Network Analysis and Engineering Directs Development and Vascularization of Multilineage Human Liver Organoids. | Velazquez, Jeremy J; LeGraw, Ryan; Moghadam, Farzaneh; Tan, Yuqi; Kilbourne, Jacquelyn; Maggiore, Joseph C; Hislop, Joshua; Liu, Silvia; Cats, Davy; Chuva de Sousa Lopes, Susana M; Plaisier, Christopher; Cahan, Patrick; Kiani, Samira; Ebrahimkhani, Mo R | Cell systems | 33290741 | 2021 Jan 20 |
Targeting de novo purine synthesis pathway via ADSL depletion impairs liver cancer growth by perturbing mitochondrial function. | Jiang, Tingting; Sánchez-Rivera, Francisco J; Soto-Feliciano, Yadira M; Yang, Qiyuan; Song, Chun-Qing; Bhuatkar, Arjun; Haynes, Cole M; Hemann, Michael T; Xue, Wen | Hepatology (Baltimore, Md.) | 33336367 | 2020 Dec 17 |
Overcoming innate immune barriers that impede AAV gene therapy vectors. | Muhuri, Manish; Maeda, Yukiko; Ma, Hong; Ram, Sanjay; Fitzgerald, Katherine A; Tai, Phillip Wl; Gao, Guangping | The Journal of clinical investigation | 33393506 | 2021 Jan 04 |
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice. | Koblan, Luke W; Erdos, Michael R; Wilson, Christopher; Cabral, Wayne A; Levy, Jonathan M; Xiong, Zheng-Mei; Tavarez, Urraca L; Davison, Lindsay M; Gete, Yantenew G; Mao, Xiaojing; Newby, Gregory A; Doherty, Sean P; Narisu, Narisu; Sheng, Quanhu; Krilow, Chad; Lin, Charles Y; Gordon, Leslie B; Cao, Kan; Collins, Francis S; Brown, Jonathan D; Liu, David R | Nature | 33408413 | 2021 Jan |
CRISPR/Cas9 gene editing for curing sickle cell disease. | Park, So Hyun; Bao, Gang | Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis | 33455878 | 2021 Jan 10 |
Precision genome editing using cytosine and adenine base editors in mammalian cells. | Huang, Tony P; Newby, Gregory A; Liu, David R | Nature protocols | 33462442 | 2021 Feb |
Determining of canine position by multiple facial landmarks to achieve natural esthetics in complete denture treatment. | Srimaneekarn, Natchalee; Arayapisit, Tawepong; Pooktuantong, Ornnicha; Cheng, Holland R; Soonsawad, Pan | The Journal of prosthetic dentistry | 33468316 | 2021 Jan 16 |
Nanoparticles for delivery of agents to fetal lungs. | Ullrich, Sarah J; Freedman-Weiss, Mollie; Ahle, Samantha; Mandl, Hanna K; Piotrowski-Daspit, Alexandra S; Roberts, Katherine; Yung, Nicholas; Maassel, Nathan; Bauer-Pisani, Tory; Ricciardi, Adele S; Egan, Marie E; Glazer, Peter M; Saltzman, W Mark; Stitelman, David H | Acta biomaterialia | 33484911 | 2021 Jan 21 |
Exercise mimetics and JAK inhibition attenuate IFN-γ-induced wasting in engineered human skeletal muscle. | Chen, Zhaowei; Li, Binjie; Zhan, Ren-Zhi; Rao, Lingjun; Bursac, Nenad | Science advances | 33523949 | 2021 Jan |
Gene-based therapies for neurodegenerative diseases. | Sun, Jichao; Roy, Subhojit | Nature neuroscience | 33526943 | 2021 03 |
Viral vector platforms within the gene therapy landscape. | Bulcha, Jote T; Wang, Yi; Ma, Hong; Tai, Phillip W L; Gao, Guangping | Signal transduction and targeted therapy | 33558455 | 2021 Feb 08 |
Soluble CD40L activates soluble and cell-surface integrins αvβ3, α5β1 and α4β1 by binding to the allosteric ligand-binding site (site 2). | Takada, Yoko K; Shimoda, Michiko; Maverakis, Emanual; Felding, Brunie H; Cheng, R Holland; Takada, Yoshikazu | The Journal of biological chemistry | 33571526 | 2021 Feb 08 |
PrimeDesign software for rapid and simplified design of prime editing guide RNAs. | Hsu, Jonathan Y; Grünewald, Julian; Szalay, Regan; Shih, Justine; Anzalone, Andrew V; Lam, Kin Chung; Shen, Max W; Petri, Karl; Liu, David R; Keith Joung, J; Pinello, Luca | Nature communications | 33589617 | 2021 02 15 |
Massively parallel assessment of human variants with base editor screens. | Hanna, Ruth E; Hegde, Mudra; Fagre, Christian R; DeWeirdt, Peter C; Sangree, Annabel K; Szegletes, Zsofia; Griffith, Audrey; Feeley, Marissa N; Sanson, Kendall R; Baidi, Yossef; Koblan, Luke W; Liu, David R; Neal, James T; Doench, John G | Cell | 33606977 | 2021 02 18 |
Engineering highly efficient backsplicing and translation of synthetic circRNAs. | Meganck, Rita M; Liu, Jiacheng; Hale, Andrew E; Simon, Katherine E; Fanous, Marco M; Vincent, Heather A; Wilusz, Jeremy E; Moorman, Nathaniel J; Marzluff, William F; Asokan, Aravind | Molecular therapy. Nucleic acids | 33614232 | 2021 Mar 05 |
COVID-19 CG enables SARS-CoV-2 mutation and lineage tracking by locations and dates of interest. | Chen, Albert Tian; Altschuler, Kevin; Zhan, Shing Hei; Chan, Yujia Alina; Deverman, Benjamin E | eLife | 33620031 | 2021 02 23 |
Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3. | Qiu, Min; Glass, Zachary; Chen, Jinjin; Haas, Mary; Jin, Xin; Zhao, Xuewei; Rui, Xuehui; Ye, Zhongfeng; Li, Yamin; Zhang, Feng; Xu, Qiaobing | Proceedings of the National Academy of Sciences of the United States of America | 33649229 | 2021 Mar 09 |
Therapeutic RNA Delivery for COVID and Other Diseases. | Dobrowolski, Curtis; Paunovska, Kalina; Hatit, Marine Z C; Lokugamage, Melissa P; Dahlman, James E | Advanced healthcare materials | 33661555 | 2021 Mar 04 |
A CRISPR-Cas9-integrase complex generates precise DNA fragments for genome integration. | Jakhanwal, Shrutee; Cress, Brady F; Maguin, Pascal; Lobba, Marco J; Marraffini, Luciano A; Doudna, Jennifer A | Nucleic acids research | 33693715 | 2021 04 06 |
Loss of sarcomeric proteins via upregulation of JAK/STAT signaling underlies interferon-γ-induced contractile deficit in engineered human myocardium. | Zhan, Ren-Zhi; Rao, Lingjun; Chen, Zhaowei; Strash, Nicholas; Bursac, Nenad | Acta biomaterialia | 33705988 | 2021 05 |
AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies. | Yang, Huiya; Brown Jr, Robert H; Wang, Dan; Strauss, Kevin A; Gao, Guangping | Trends in molecular medicine | 33714697 | 2021 06 |
Tissue-Engineered Skeletal Muscle Models to Study Muscle Function, Plasticity, and Disease. | Khodabukus, Alastair | Frontiers in physiology | 33716768 | 2021 |
Multicellular Systems to Translate Somatic Cell Genome Editors to Humans. | Hernandez-Gordillo, Victor; Casolaro, Thomas Caleb; Ebrahimkhani, Mo R; Kiani, Samira | Current opinion in biomedical engineering | 33718690 | 2020 Dec |
Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression. | Gao, Pan; Lyu, Qing; Ghanam, Amr R; Lazzarotto, Cicera R; Newby, Gregory A; Zhang, Wei; Choi, Mihyun; Slivano, Orazio J; Holden, Kevin; Walker 2nd, John A; Kadina, Anastasia P; Munroe, Rob J; Abratte, Christian M; Schimenti, John C; Liu, David R; Tsai, Shengdar Q; Long, Xiaochun; Miano, Joseph M | Genome biology | 33722289 | 2021 03 16 |
SARS-CoV-2 infection of human iPSC-derived cardiac cells reflects cytopathic features in hearts of patients with COVID-19. | Perez-Bermejo, Juan A; Kang, Serah; Rockwood, Sarah J; Simoneau, Camille R; Joy, David A; Silva, Ana C; Ramadoss, Gokul N; Flanigan, Will R; Fozouni, Parinaz; Li, Huihui; Chen, Pei-Yi; Nakamura, Ken; Whitman, Jeffrey D; Hanson, Paul J; McManus, Bruce M; Ott, Melanie; Conklin, Bruce R; McDevitt, Todd C | Science translational medicine | 33723017 | 2021 04 21 |
Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligands. | Gan, Zubao; Lokugamage, Melissa P; Hatit, Marine Z C; Loughrey, David; Paunovska, Kalina; Sato, Manaka; Cristian, Ana; Dahlman, James E | Bioengineering & translational medicine | 33758781 | 2020 Sep |
Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy. | Marquez Loza, Laura I; Cooney, Ashley L; Dong, Qian; Randak, Christoph O; Rivella, Stefano; Sinn, Patrick L; McCray Jr, Paul B | Molecular therapy. Methods & clinical development | 33768133 | 2021 Jun 11 |
Large-scale molecular epidemiological analysis of AAV in a cancer patient population. | Qin, Wanru; Xu, Guangchao; Tai, Phillip W L; Wang, Chunmei; Luo, Li; Li, Chengjian; Hu, Xun; Xue, Jianxin; Lu, You; Zhou, Qiao; Wei, Qiang; Wen, Tianfu; Hu, Jiankun; Xiao, Yuanyuan; Yang, Li; Li, Weimin; Flotte, Terence R; Wei, Yuquan; Gao, Guangping | Oncogene | 33782545 | 2021 Apr |
PEGylation of poly(amine-co-ester) polyplexes for tunable gene delivery. | Grun, Molly K; Suberi, Alexandra; Shin, Kwangsoo; Lee, Teresa; Gomerdinger, Victoria; Moscato, Zoe M; Piotrowski-Daspit, Alexandra S; Saltzman, W Mark | Biomaterials | 33813260 | 2021 May |
Examining the evidence for extracellular RNA function in mammals. | Gruner, Hannah N; McManus, Michael T | Nature reviews. Genetics | 33824487 | 2021 07 |
The NIH Somatic Cell Genome Editing program. | Saha, Krishanu; Sontheimer, Erik J; Brooks, P J; Dwinell, Melinda R; Gersbach, Charles A; Liu, David R; Murray, Stephen A; Tsai, Shengdar Q; Wilson, Ross C; Anderson, Daniel G; Asokan, Aravind; Banfield, Jillian F; Bankiewicz, Krystof S; Bao, Gang; Bulte, Jeff W M; Bursac, Nenad; Campbell, Jarryd M; Carlson, Daniel F; Chaikof, Elliot L; Chen, Zheng-Yi; Cheng, R Holland; Clark, Karl J; Curiel, David T; Dahlman, James E; Deverman, Benjamin E; Dickinson, Mary E; Doudna, Jennifer A; Ekker, Stephen C; Emborg, Marina E; Feng, Guoping; Freedman, Benjamin S; Gamm, David M; Gao, Guangping; Ghiran, Ionita C; Glazer, Peter M; Gong, Shaoqin; Heaney, Jason D; Hennebold, Jon D; Hinson, John T; Khvorova, Anastasia; Kiani, Samira; Lagor, William R; Lam, Kit S; Leong, Kam W; Levine, Jon E; Lewis, Jennifer A; Lutz, Cathleen M; Ly, Danith H; Maragh, Samantha; McCray Jr, Paul B; McDevitt, Todd C; Mirochnitchenko, Oleg; Morizane, Ryuji; Murthy, Niren; Prather, Randall S; Ronald, John A; Roy, Subhojit; Roy, Sushmita; Sabbisetti, Venkata; Saltzman, W Mark; Santangelo, Philip J; Segal, David J; Shimoyama, Mary; Skala, Melissa C; Tarantal, Alice F; Tilton, John C; Truskey, George A; Vandsburger, Moriel; Watts, Jonathan K; Wells, Kevin D; Wolfe, Scot A; Xu, Qiaobing; Xue, Wen; Yi, Guohua; Zhou, Jiangbing; SCGE Consortium | Nature | 33828315 | 2021 Apr |
Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice. | Liu, Pengpeng; Liang, Shun-Qing; Zheng, Chunwei; Mintzer, Esther; Zhao, Yan G; Ponnienselvan, Karthikeyan; Mir, Aamir; Sontheimer, Erik J; Gao, Guangping; Flotte, Terence R; Wolfe, Scot A; Xue, Wen | Nature communications | 33837189 | 2021 04 09 |
VectorMOD: Method for Bottom-Up Proteomic Characterization of rAAV Capsid Post-Translational Modifications and Vector Impurities. | Rumachik, Neil G; Malaker, Stacy A; Paulk, Nicole K | Frontiers in immunology | 33868302 | 2021 |
Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science. | Abdeen, Amr A; Cosgrove, Brian D; Gersbach, Charles A; Saha, Krishanu | Annual review of biomedical engineering | 33909475 | 2021 07 13 |
Sarcomere function activates a p53-dependent DNA damage response that promotes polyploidization and limits in vivo cell engraftment. | Pettinato, Anthony M; Yoo, Dasom; VanOudenhove, Jennifer; Chen, Yu-Sheng; Cohn, Rachel; Ladha, Feria A; Yang, Xiulan; Thakar, Ketan; Romano, Robert; Legere, Nicolas; Meredith, Emily; Robson, Paul; Regnier, Michael; Cotney, Justin L; Murry, Charles E; Hins | Cell reports | 33951429 | 2021 May 04 |
Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease. | Wang, Jason; Zhou, Chris J; Khodabukus, Alastair; Tran, Sabrina; Han, Sang-Oh; Carlson, Aaron L; Madden, Lauran; Kishnani, Priya S; Koeberl, Dwight D; Bursac, Nenad | Communications biology | 33953320 | 2021 05 05 |
3D cell culture models: Drug pharmacokinetics, safety assessment, and regulatory consideration. | Wang, Hongbing; Brown, Paul C; Chow, Edwin C Y; Ewart, Lorna; Ferguson, Stephen S; Fitzpatrick, Suzanne; Freedman, Benjamin S; Guo, Grace L; Hedrich, William; Heyward, Scott; Hickman, James; Isoherranen, Nina; Li, Albert P; Liu, Qi; Mumenthaler, Shannon M; Polli, James; Proctor, William R; Ribeiro, Alexandre; Wang, Jian-Ying; Wange, Ronald L; Huang, Shiew-Mei | Clinical and translational science | 33982436 | 2021 09 |
Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction. | Muhuri, Manish; Zhan, Wei; Maeda, Yukiko; Li, Jia; Lotun, Anoushka; Chen, Jennifer; Sylvia, Katelyn; Dasgupta, Ishani; Arjomandnejad, Motahareh; Nixon, Thomas; Keeler, Allison M; Manokaran, Sangeetha; He, Ran; Su, Qin; Tai, Phillip W L; Gao, Guangping | Frontiers in immunology | 33995418 | 2021 |
Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repair. | Wilde, Jonathan J; Aida, Tomomi; Del Rosario, Ricardo C H; Kaiser, Tobias; Qi, Peimin; Wienisch, Martin; Zhang, Qiangge; Colvin, Steven; Feng, Guoping | Cell | 34043941 | 2021 Jun 10 |
Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens? | Zhan, Wei; Muhuri, Manish; Tai, Phillip W L; Gao, Guangping | Frontiers in immunology | 34046041 | 2021 |
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Electrophoretic mobility shift as a molecular beacon-based readout for miRNA detection. | Oliveira-Jr, Getulio P; Barbosa, Raquel H; Thompson, Lauren; Pinckney, Brandy; Murphy-Thornley, Moriah; Lu, Shulin; Jones, Jennifer; Hansen, Clinton H; Tigges, John; Wong, Wesley P; Ghiran, Ionita C | Biosensors & bioelectronics | 34062334 | 2021 Oct 01 |
Generation and Genetic Correction of USH2A c.2299delG Mutation in Patient-Derived Induced Pluripotent Stem Cells. | Liu, Xuezhong; Lillywhite, Justin; Zhu, Wenliang; Huang, Zaohua; Clark, Anna M; Gosstola, Nicholas; Maguire, Colin T; Dykxhoorn, Derek; Chen, Zheng-Yi; Yang, Jun | Genes | 34070435 | 2021 05 25 |
SARS-CoV-2 infection induces the activation of tissue factor-mediated coagulation via activation of acid sphingomyelinase. | Wang, Jue; Pendurthi, Usha R; Yi, Guohua; Rao, L Vijaya Mohan | Blood | 34075401 | 2021 07 29 |
Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering. | Hamilton, Jennifer R; Tsuchida, Connor A; Nguyen, David N; Shy, Brian R; McGarrigle, E Riley; Sandoval Espinoza, Cindy R; Carr, Daniel; Blaeschke, Franziska; Marson, Alexander; Doudna, Jennifer A | Cell reports | 34077734 | 2021 Jun 01 |
Escaping the endosome: assessing cellular trafficking mechanisms of non-viral vehicles. | Xu, Emily; Saltzman, W Mark; Piotrowski-Daspit, Alexandra S | Journal of controlled release : official journal of the Controlled Release Society | 34077782 | 2021 07 10 |
Base editing of haematopoietic stem cells rescues sickle cell disease in mice. | Newby, Gregory A; Yen, Jonathan S; Woodard, Kaitly J; Mayuranathan, Thiyagaraj; Lazzarotto, Cicera R; Li, Yichao; Sheppard-Tillman, Heather; Porter, Shaina N; Yao, Yu; Mayberry, Kalin; Everette, Kelcee A; Jang, Yoonjeong; Podracky, Christopher J; Thaman, Elizabeth; Lechauve, Christophe; Sharma, Akshay; Henderson, Jordana M; Richter, Michelle F; Zhao, Kevin T; Miller, Shannon M; Wang, Tina; Koblan, Luke W; McCaffrey, Anton P; Tisdale, John F; Kalfa, Theodosia A; Pruett-Miller, Shondra M; Tsai, Shengdar Q; Weiss, Mitchell J; Liu, David R | Nature | 34079130 | 2021 Jul |
Mechanisms of angiogenic incompetence in Hutchinson-Gilford progeria via downregulation of endothelial NOS. | Gete, Yantenew G; Koblan, Luke W; Mao, Xiaojing; Trappio, Mason; Mahadik, Bhushan; Fisher, John P; Liu, David R; Cao, Kan | Aging cell | 34086398 | 2021 07 |
Targeting the Apoa1 locus for liver-directed gene therapy. | De Giorgi, Marco; Li, Ang; Hurley, Ayrea; Barzi, Mercedes; Doerfler, Alexandria M; Cherayil, Nikitha A; Smith, Harrison E; Brown, Jonathan D; Lin, Charles Y; Bissig, Karl-Dimiter; Bao, Gang; Lagor, William R | Molecular therapy. Methods & clinical development | 34141821 | 2021 Jun 11 |
Magnetic Forces Enable Control of Biological Processes In Vivo. | Bao, Gang | Journal of applied mechanics | 34168385 | 2021 Mar 01 |
In vivo targeted delivery of nucleic acids and CRISPR genome editors enabled by GSH-responsive silica nanoparticles. | Wang, Yuyuan; Shahi, Pawan K; Wang, Xiuxiu; Xie, Ruosen; Zhao, Yi; Wu, Min; Roge, Seth; Pattnaik, Bikash R; Gong, Shaoqin | Journal of controlled release : official journal of the Controlled Release Society | 34174352 | 2021 Jun 23 |
Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques. | AlJanahi, Aisha A; Lazzarotto, Cicera R; Chen, Shirley; Shin, Tae-Hoon; Cordes, Stefan; Fan, Xing; Jabara, Isabel; Zhou, Yifan; Young, David J; Lee, Byung-Chul; Yu, Kyung-Rok; Li, Yuesheng; Toms, Bradley; Tunc, Ilker; Hong, So Gun; Truitt, Lauren L; Klermund, Julia; Andrieux, Geoffroy; Kim, Miriam Y; Cathomen, Toni; Gill, Saar; Tsai, Shengdar Q; Dunbar, Cynthia E | Molecular therapy : the journal of the American Society of Gene Therapy | 34174439 | 2022 01 05 |
Controlling CRISPR with small molecule regulation for somatic cell genome editing. | Khajanchi, Namita; Saha, Krishanu | Molecular therapy : the journal of the American Society of Gene Therapy | 34174442 | 2021 Jun 24 |
Gene editing in a Myo6 semi-dominant mouse model rescues auditory function. | Xue, Yuanyuan; Hu, Xinde; Wang, Daqi; Li, Di; Li, Yige; Wang, Fang; Huang, Mingqian; Gu, Xi; Xu, Zhijiao; Zhou, Jinan; Wang, Jinghan; Chai, Renjie; Shen, Jun; Chen, Zheng-Yi; Li, Geng-Lin; Yang, Hui; Li, Huawei; Zuo, Erwei; Shu, Yilai | Molecular therapy : the journal of the American Society of Gene Therapy | 34174443 | 2021 Jun 24 |
Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning. | Koblan, Luke W; Arbab, Mandana; Shen, Max W; Hussmann, Jeffrey A; Anzalone, Andrew V; Doman, Jordan L; Newby, Gregory A; Yang, Dian; Mok, Beverly; Replogle, Joseph M; Xu, Albert; Sisley, Tyler A; Weissman, Jonathan S; Adamson, Britt; Liu, David R | Nature biotechnology | 34183861 | 2021 Jun 28 |
Mucopolysaccharidoses type I gene therapy. | Hurt, Sarah C; Dickson, Patricia I; Curiel, David T | Journal of inherited metabolic disease | 34189746 | 2021 Jun 29 |
rAAV-based and intraprostatically delivered miR-34a therapeutics for efficient inhibition of prostate cancer progression. | Ai, Jianzhong; Li, Jia; Su, Qin; Ma, Hong; He, Ran; Wei, Qiang; Li, Hong; Gao, Guangping | Gene therapy | 34226687 | 2021 Jul 06 |
Receptor Switching in Newly Evolved Adeno-associated Viruses. | Havlik, L Patrick; Das, Anshuman; Mietzsch, Mario; Oh, Daniel K; Ark, Jonathan; McKenna, Robert; Agbandje-McKenna, Mavis; Asokan, Aravind | Journal of virology | 34232726 | 2021 09 09 |
Recent innovations in fluorescence lifetime imaging microscopy for biology and medicine. | Datta, Rupsa; Gillette, Amani; Stefely, Matthew; Skala, Melissa C | Journal of biomedical optics | 34247457 | 2021 07 |
External stimuli-responsive nanoparticles for spatially and temporally controlled delivery of CRISPR-Cas genome editors. | Xie, Ruosen; Wang, Yuyuan; Gong, Shaoqin | Biomaterials science | 34286726 | 2021 Sep 14 |
Vector Strategies to Actualize B Cell-Based Gene Therapies. | Jeske, Amanda M; Boucher, Paul; Curiel, David T; Voss, James E | Journal of immunology (Baltimore, Md. : 1950) | 34321286 | 2021 08 01 |
Label-free imaging for quality control of cardiomyocyte differentiation. | Qian, Tongcheng; Heaster, Tiffany M; Houghtaling, Angela R; Sun, Kexin; Samimi, Kayvan; Skala, Melissa C | Nature communications | 34321477 | 2021 07 28 |
Transgenic mice for in vivo epigenome editing with CRISPR-based systems. | Gemberling, Matthew P; Siklenka, Keith; Rodriguez, Erica; Tonn-Eisinger, Katherine R; Barrera, Alejandro; Liu, Fang; Kantor, Ariel; Li, Liqing; Cigliola, Valentina; Hazlett, Mariah F; Williams, Courtney A; Bartelt, Luke C; Madigan, Victoria J; Bodle, Josephine C; Daniels, Heather; Rouse, Douglas C; Hilton, Isaac B; Asokan, Aravind; Ciofani, Maria; Poss, Kenneth D; Reddy, Timothy E; West, Anne E; Gersbach, Charles A | Nature methods | 34341582 | 2021 08 |
Actinin BioID reveals sarcomere crosstalk with oxidative metabolism through interactions with IGF2BP2. | Ladha, Feria A; Thakar, Ketan; Pettinato, Anthony M; Legere, Nicholas; Ghahremani, Shahnaz; Cohn, Rachel; Romano, Robert; Meredith, Emily; Chen, Yu-Sheng; Hinson, J Travis | Cell reports | 34380038 | 2021 Aug 10 |
DNA interference states of the hypercompact CRISPR-CasΦ effector. | Pausch, Patrick; Soczek, Katarzyna M; Herbst, Dominik A; Tsuchida, Connor A; Al-Shayeb, Basem; Banfield, Jillian F; Nogales, Eva; Doudna, Jennifer A | Nature structural & molecular biology | 34381246 | 2021 08 |
A Versatile and Robust Platform for the Scalable Manufacture of Biomimetic Nanovaccines. | Hu, Hanze; Yang, Chao; Zhang, Fan; Li, Mingqiang; Tu, Zhaoxu; Mu, Lizhong; Dawulieti, Jianati; Lao, Yeh-Hsing; Xiao, Zixuan; Yan, Huize; Sun, Wen; Shao, Dan; Leong, Kam W | Advanced science (Weinheim, Baden-Wurttemberg, Germany) | 34386315 | 2021 08 |
Bioengineered optogenetic model of human neuromuscular junction. | Vila, Olaia F; Chavez, Miguel; Ma, Stephen P; Yeager, Keith; Zholudeva, Lyandysha V; Colón-Mercado, Jennifer M; Qu, Yihuai; Nash, Trevor R; Lai, Carmen; Feliciano, Carissa M; Carter, Matthew; Kamm, Roger D; Judge, Luke M; Conklin, Bruce R; Ward, Michael E; McDevitt, Todd C; Vunjak-Novakovic, Gordana | Biomaterials | 34403849 | 2021 09 |
Easy-Prime: a machine learning-based prime editor design tool. | Li, Yichao; Chen, Jingjing; Tsai, Shengdar Q; Cheng, Yong | Genome biology | 34412673 | 2021 08 19 |
Synthetic Biology Approaches for Engineering Next-Generation Adenoviral Gene Therapies. | Collins, Logan Thrasher; Curiel, David T | ACS nano | 34415739 | 2021 09 28 |
Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical Applications. | Hu, Hanze; Yang, Chao; Li, Mingqiang; Shao, Dan; Mao, Hai-Quan; Leong, Kam W | Materials today (Kidlington, England) | 34421329 | 2021 Jan-Feb |
Cortical overgrowth in a preclinical forebrain organoid model of CNTNAP2-associated autism spectrum disorder. | de Jong, Job O; Llapashtica, Ceyda; Genestine, Matthieu; Strauss, Kevin; Provenzano, Frank; Sun, Yan; Zhu, Huixiang; Cortese, Giuseppe P; Brundu, Francesco; Brigatti, Karlla W; Corneo, Barbara; Migliori, Bianca; Tomer, Raju; Kushner, Steven A; Kellendonk, Christoph; Javitch, Jonathan A; Xu, Bin; Markx, Sander | Nature communications | 34471112 | 2021 09 01 |
Light: A Magical Tool for Controlled Drug Delivery. | Tao, Yu; Chan, Hon Fai; Shi, Bingyang; Li, Mingqiang; Leong, Kam W | Advanced functional materials | 34483808 | 2020 Dec 01 |
Allele-Specific Gene Editing Rescues Pathology in a Human Model of Charcot-Marie-Tooth Disease Type 2E. | Feliciano, Carissa M; Wu, Kenneth; Watry, Hannah L; Marley, Chiara B E; Ramadoss, Gokul N; Ghanim, Hana Y; Liu, Angela Z; Zholudeva, Lyandysha V; McDevitt, Todd C; Saporta, Mario A; Conklin, Bruce R; Judge, Luke M | Frontiers in cell and developmental biology | 34485306 | 2021 |
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy. | Pickar-Oliver, Adrian; Gough, Veronica; Bohning, Joel D; Liu, Siyan; Robinson-Hamm, Jacqueline N; Daniels, Heather; Majoros, William H; Devlin, Garth; Asokan, Aravind; Gersbach, Charles A | Molecular therapy : the journal of the American Society of Gene Therapy | 34509668 | 2021 Nov 03 |
In vivo somatic cell base editing and prime editing. | Newby, Gregory A; Liu, David R | Molecular therapy : the journal of the American Society of Gene Therapy | 34509669 | 2021 Nov 03 |
Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors. | Krishnamurthy, Sateesh; Traore, Soumba; Cooney, Ashley L; Brommel, Christian M; Kulhankova, Katarina; Sinn, Patrick L; Newby, Gregory A; Liu, David R; McCray, Paul B | Nucleic acids research | 34520545 | 2021 Oct 11 |
In Vivo MRI Tracking of Tumor Vaccination and Antigen Presentation by Dendritic Cells. | Bulte, Jeff W M; Shakeri-Zadeh, Ali | Molecular imaging and biology | 34581954 | 2021 Sep 28 |
Engineered pegRNAs improve prime editing efficiency. | Nelson, James W; Randolph, Peyton B; Shen, Simon P; Everette, Kelcee A; Chen, Peter J; Anzalone, Andrew V; An, Meirui; Newby, Gregory A; Chen, Jonathan C; Hsu, Alvin; Liu, David R | Nature biotechnology | 34608327 | 2021 Oct 04 |
Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs. | Lokugamage, Melissa P; Vanover, Daryll; Beyersdorf, Jared; Hatit, Marine Z C; Rotolo, Laura; Echeverri, Elisa Schrader; Peck, Hannah E; Ni, Huanzhen; Yoon, Jeong-Kee; Kim, YongTae; Santangelo, Philip J; Dahlman, James E | Nature biomedical engineering | 34616046 | 2021 09 |
Kidney development to kidney organoids and back again. | Gupta, Navin; Morizane, Ryuji | Seminars in cell & developmental biology | 34627669 | 2021 Oct 06 |
Surface Topography of Polyethylene Glycol Shell Nanoparticles Formed from Bottlebrush Block Copolymers Controls Interactions with Proteins and Cells. | Grundler, Julian; Shin, Kwangsoo; Suh, Hee-Won; Zhong, Mingjiang; Saltzman, W Mark | ACS nano | 34633171 | 2021 10 26 |
Disulfide-compatible phage-assisted continuous evolution in the periplasmic space. | Morrison, Mary S; Wang, Tina; Raguram, Aditya; Hemez, Colin; Liu, David R | Nature communications | 34645844 | 2021 10 13 |
Deletion and replacement of long genomic sequences using prime editing. | Jiang, Tingting; Zhang, Xiao-Ou; Weng, Zhiping; Xue, Wen | Nature biotechnology | 34650270 | 2021 Oct 14 |
Enhanced prime editing systems by manipulating cellular determinants of editing outcomes. | Chen, Peter J; Hussmann, Jeffrey A; Yan, Jun; Knipping, Friederike; Ravisankar, Purnima; Chen, Pin-Fang; Chen, Cidi; Nelson, James W; Newby, Gregory A; Sahin, Mustafa; Osborn, Mark J; Weissman, Jonathan S; Adamson, Britt; Liu, David R | Cell | 34653350 | 2021 Oct 28 |
SnRNA sequencing defines signaling by RBC-derived extracellular vesicles in the murine heart. | Valkov, Nedyalka; Das, Avash; Tucker, Nathan R; Li, Guoping; Salvador, Ane M; Chaffin, Mark D; Pereira De Oliveira Junior, Getulio; Kur, Ivan; Gokulnath, Priyanka; Ziegler, Olivia; Yeri, Ashish; Lu, Shulin; Khamesra, Aushee; Xiao, Chunyang; Rodosthenous, | Life science alliance | 34663679 | 2021 Dec |
5'-Modifications improve potency and efficacy of DNA donors for precision genome editing. | Ghanta, Krishna S; Chen, Zexiang; Mir, Aamir; Dokshin, Gregoriy A; Krishnamurthy, Pranathi M; Yoon, Yeonsoo; Gallant, Judith; Xu, Ping; Zhang, Xiao-Ou; Ozturk, Ahmet Rasit; Shin, Masahiro; Idrizi, Feston; Liu, Pengpeng; Gneid, Hassan; Edraki, Alireza; Lawson, Nathan D; Rivera-Pérez, Jaime A; Sontheimer, Erik J; Watts, Jonathan K; Mello, Craig C | eLife | 34665130 | 2021 10 19 |
Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome Editing. | Qiu, Min; Li, Yamin; Bloomer, Hanan; Xu, Qiaobing | Accounts of chemical research | 34668716 | 2021 11 02 |
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway. | Liang, Shun-Qing; Walkey, Christopher J; Martinez, Alexa E; Su, Qin; Dickinson, Mary E; Wang, Dan; Lagor, William R; Heaney, Jason D; Gao, Guangping; Xue, Wen | Molecular therapy : the journal of the American Society of Gene Therapy | 34695545 | 2021 Oct 23 |
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo. | Ibraheim, Raed; Tai, Phillip W L; Mir, Aamir; Javeed, Nida; Wang, Jiaming; Rodríguez, Tomás C; Namkung, Suk; Nelson, Samantha; Khokhar, Eraj Shafiq; Mintzer, Esther; Maitland, Stacy; Chen, Zexiang; Cao, Yueying; Tsagkaraki, Emmanouela; Wolfe, Scot A; Wang, Dan; Pai, Athma A; Xue, Wen; Gao, Guangping; Sontheimer, Erik J | Nature communications | 34725353 | 2021 11 01 |
Gain-of-function cardiomyopathic mutations in RBM20 rewire splicing regulation and re-distribute ribonucleoprotein granules within processing bodies. | Fenix, Aidan M; Miyaoka, Yuichiro; Bertero, Alessandro; Blue, Steven M; Spindler, Matthew J; Tan, Kenneth K B; Perez-Bermejo, Juan A; Chan, Amanda H; Mayerl, Steven J; Nguyen, Trieu D; Russell, Caitlin R; Lizarraga, Paweena P; Truong, Annie; So, Po-Lin; Kulkarni, Aishwarya; Chetal, Kashish; Sathe, Shashank; Sniadecki, Nathan J; Yeo, Gene W; Murry, Charles E; Conklin, Bruce R; Salomonis, Nathan | Nature communications | 34732726 | 2021 11 03 |
Disruption of HIV-1 co-receptors CCR5 and CXCR4 in primary human T cells and hematopoietic stem and progenitor cells using base editing. | Knipping, Friederike; Newby, Gregory A; Eide, Cindy R; McElroy, Amber N; Nielsen, Sarah C; Smith, Kyle; Fang, Yongxing; Cornu, Tatjana I; Costa, Caroline; Gutierrez-Guerrero, Alejandra; Bingea, Samuel P; Feser, Colby J; Steinbeck, Benjamin; Hippen, Keli L; Blazar, Bruce R; McCaffrey, Anton; Mussolino, Claudio; Verhoeyen, Els; Tolar, Jakub; Liu, David R; Osborn, Mark J | Molecular therapy : the journal of the American Society of Gene Therapy | 34737067 | 2022 01 05 |
Cross-validation of SARS-CoV-2 responses in kidney organoids and clinical populations. | Helms, Louisa; Marchiano, Silvia; Stanaway, Ian B; Hsiang, Tien-Ying; Juliar, Benjamin A; Saini, Shally; Zhao, Yan Ting; Khanna, Akshita; Menon, Rajasree; Alakwaa, Fadhl; Mikacenic, Carmen; Morrell, Eric D; Wurfel, Mark M; Kretzler, Matthias; Harder, Jennifer L; Murry, Charles E; Himmelfarb, Jonathan; Ruohola-Baker, Hannele; Bhatraju, Pavan K; Gale Jr, Michael; Freedman, Benjamin S | JCI insight | 34767537 | 2021 12 22 |
Defining genome-wide CRISPR-Cas genome-editing nuclease activity with GUIDE-seq. | Malinin, Nikolay L; Lee, GaHyun; Lazzarotto, Cicera R; Li, Yichao; Zheng, Zongli; Nguyen, Nhu T; Liebers, Matthew; Topkar, Ved V; Iafrate, A John; Le, Long P; Aryee, Martin J; Joung, J Keith; Tsai, Shengdar Q | Nature protocols | 34773119 | 2021 Nov 12 |
Neuromuscular Development and Disease: Learning From in vitro and in vivo Models. | Fralish, Zachary; Lotz, Ethan M; Chavez, Taylor; Khodabukus, Alastair; Bursac, Nenad | Frontiers in cell and developmental biology | 34778273 | 2021 |
Synthetic biology: at the crossroads of genetic engineering and human therapeutics-a Keystone Symposia report. | Cable, Jennifer; Leonard, Joshua N; Lu, Timothy K; Xie, Zhen; Chang, Matthew Wook; Fernández, Luis Ángel; Lora, José M; Kaufman, Howard L; Quintana, Francisco J; Geiger, Roger; F Lesser, Cammie; Lynch, Jason P; Hava, David L; Cornish, Virginia W; Lee, Gary K; DiAndreth, Breanna; Fero, Michael; Srivastava, Rajkamal; De Coster, Tim; Roybal, Kole T; Rackham, Owen J L; Kiani, Samira; Zhu, Iowis; Hernandez-Lopez, Rogelio A; Guo, Tingxi; Chen, William C W | Annals of the New York Academy of Sciences | 34786712 | 2021 12 |
Deleting DNMT3A in CAR T cells prevents exhaustion and enhances antitumor activity. | Prinzing, Brooke; Zebley, Caitlin C; Petersen, Christopher T; Fan, Yiping; Anido, Alejandro Allo; Yi, Zhongzhen; Nguyen, Phuong; Houke, Haley; Bell, Matthew; Haydar, Dalia; Brown, Charmaine; Boi, Shannon K; Alli, Shanta; Crawford, Jeremy Chase; Riberdy, Janice M; Park, Jeoungeun J; Zhou, Sheng; Velasquez, Mireya Paulina; DeRenzo, Chris; Lazzarotto, Cicera R; Tsai, Shengdar Q; Vogel, Peter; Pruett-Miller, Shondra M; Langfitt, Deanna M; Gottschalk, Stephen; Youngblood, Ben; Krenciute, Giedre | Science translational medicine | 34788079 | 2021 Nov 17 |
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. | Hakim, Chady H; Kumar, Sandeep R P; Pérez-López, Dennis O; Wasala, Nalinda B; Zhang, Dong; Yue, Yongping; Teixeira, James; Pan, Xiufang; Zhang, Keqing; Million, Emily D; Nelson, Christopher E; Metzger, Samantha; Han, Jin; Louderman, Jacqueline A; Schmidt, Florian; Feng, Feng; Grimm, Dirk; Smith, Bruce F; Yao, Gang; Yang, N Nora; Gersbach, Charles A; Chen, Shi-Jie; Herzog, Roland W; Duan, Dongsheng | Nature communications | 34819506 | 2021 11 24 |
CRISPR-enhanced human adipocyte browning as cell therapy for metabolic disease. | Tsagkaraki, Emmanouela; Nicoloro, Sarah M; DeSouza, Tiffany; Solivan-Rivera, Javier; Desai, Anand; Lifshitz, Lawrence M; Shen, Yuefei; Kelly, Mark; Guilherme, Adilson; Henriques, Felipe; Amrani, Nadia; Ibraheim, Raed; Rodriguez, Tomas C; Luk, Kevin; Maitland, Stacy; Friedline, Randall H; Tauer, Lauren; Hu, Xiaodi; Kim, Jason K; Wolfe, Scot A; Sontheimer, Erik J; Corvera, Silvia; Czech, Michael P | Nature communications | 34836963 | 2021 11 26 |
The FusX TALE Base Editor (FusXTBE) for Rapid Mitochondrial DNA Programming of Human Cells In Vitro and Zebrafish Disease Models In Vivo. | Sabharwal, Ankit; Kar, Bibekananda; Restrepo-Castillo, Santiago; Holmberg, Shannon R; Mathew, Neal D; Kendall, Benjamin Luke; Cotter, Ryan P; WareJoncas, Zachary; Seiler, Christoph; Nakamaru-Ogiso, Eiko; Clark, Karl J; Ekker, Stephen C | The CRISPR journal | 34847747 | 2021 12 |
The Coiled-Coil Forming Peptide (KVSALKE)5 Is a Cell Penetrating Peptide that Enhances the Intracellular Delivery of Proteins. | Li, Jie; Tuma, Jan; Han, Hesong; Kim, Hansol; Wilson, Ross C; Lee, Hye Young; Murthy, Niren | Advanced healthcare materials | 34861744 | 2021 Dec 03 |
Author Correction: Engineered pegRNAs improve prime editing efficiency. | Nelson, James W; Randolph, Peyton B; Shen, Simon P; Everette, Kelcee A; Chen, Peter J; Anzalone, Andrew V; An, Meirui; Newby, Gregory A; Chen, Jonathan C; Hsu, Alvin; Liu, David R | Nature biotechnology | 34880468 | 2021 Dec 08 |
Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing. | Anzalone, Andrew V; Gao, Xin D; Podracky, Christopher J; Nelson, Andrew T; Koblan, Luke W; Raguram, Aditya; Levy, Jonathan M; Mercer, Jaron A M; Liu, David R | Nature biotechnology | 34887556 | 2021 Dec 09 |
AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset. | Goertsen, David; Flytzanis, Nicholas C; Goeden, Nick; Chuapoco, Miguel R; Cummins, Alexander; Chen, Yijing; Fan, Yingying; Zhang, Qiangge; Sharma, Jitendra; Duan, Yangyang; Wang, Liping; Feng, Guoping; Chen, Yu; Ip, Nancy Y; Pickel, James; Gradinaru, Viviana | Nature neuroscience | 34887588 | 2022 01 |
Breast cancer immunotherapy: current biomarkers and the potential of in vitro assays. | Skala, Melissa C; Ayuso, Jose M; Burkard, Mark E; Deming, Dustin A | Current opinion in biomedical engineering | 34901585 | 2021 Mar |
Reading Frame Repair of TTN Truncation Variants Restores Titin Quantity and Functions. | Romano, Robert; Ghahremani, Shahnaz; Zimmerman, Talia; Legere, Nicholas; Thakar, Ketan; Ladha, Feria A; Pettinato, Anthony M; Hinson, J Travis | Circulation | 34905694 | 2022 01 18 |
Acid-Sensitive Surfactants Enhance the Delivery of Nucleic Acids. | Røise, Joachim Justad; Han, Hesong; Li, Jie; Kerr, D Lucas; Taing, Chung; Behrouzi, Kamyar; He, Maomao; Ruan, Emily; Chan, Lienna Y; Espinoza, Eli M; Reinhard, Sören; Thakker, Kanav; Kwon, Justin; Mofrad, Mohammad R K; Murthy, Niren | Molecular pharmaceutics | 34931518 | 2022 01 03 |
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rAAV-delivered PTEN therapeutics for prostate cancer. | Ai, Jianzhong; Li, Jia; Su, Qin; Ma, Hong; Wei, Qiang; Li, Hong; Gao, Guangping | Molecular therapy. Nucleic acids | 34976432 | 2022 Mar 08 |
Editorial: "AAV Gene Therapy: Immunology and Immunotherapeutics". | Martinez-Navio, José M; Paulk, Nicole K; Gao, Guangping | Frontiers in immunology | 34992614 | 2021 |
A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver. | Zheng, Chunwei; Liang, Shun-Qing; Liu, Bin; Liu, Pengpeng; Kwan, Suet-Yan; Wolfe, Scot A; Xue, Wen | Molecular therapy : the journal of the American Society of Gene Therapy | 34998953 | 2022 Jan 05 |
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins. | Banskota, Samagya; Raguram, Aditya; Suh, Susie; Du, Samuel W; Davis, Jessie R; Choi, Elliot H; Wang, Xiao; Nielsen, Sarah C; Newby, Gregory A; Randolph, Peyton B; Osborn, Mark J; Musunuru, Kiran; Palczewski, Krzysztof; Liu, David R | Cell | 35021064 | 2022 01 20 |
BRD2 inhibition blocks SARS-CoV-2 infection by reducing transcription of the host cell receptor ACE2. | Samelson, Avi J; Tran, Quang Dinh; Robinot, Rémy; Carrau, Lucia; Rezelj, Veronica V; Kain, Alice Mac; Chen, Merissa; Ramadoss, Gokul N; Guo, Xiaoyan; Lim, Shion A; Lui, Irene; Nuñez, James K; Rockwood, Sarah J; Wang, Jianhui; Liu, Na; Carlson-Stevermer, Jared; Oki, Jennifer; Maures, Travis; Holden, Kevin; Weissman, Jonathan S; Wells, James A; Conklin, Bruce R; TenOever, Benjamin R; Chakrabarti, Lisa A; Vignuzzi, Marco; Tian, Ruilin; Kampmann, Martin | Nature cell biology | 35027731 | 2022 01 |
Detecting Cardiovascular Protein-Protein Interactions by Proximity Proteomics. | Circulation research | 35050691 | 2022 01 21 | |
Genome-wide detection of CRISPR editing in vivo using GUIDE-tag. | Liang, Shun-Qing; Liu, Pengpeng; Smith, Jordan L; Mintzer, Esther; Maitland, Stacy; Dong, Xiaolong; Yang, Qiyuan; Lee, Jonathan; Haynes, Cole M; Zhu, Lihua Julie; Watts, Jonathan K; Sontheimer, Erik J; Wolfe, Scot A; Xue, Wen | Nature communications | 35064134 | 2022 01 21 |
pH-Responsive Polymer Nanoparticles for Efficient Delivery of Cas9 Ribonucleoprotein With or Without Donor DNA. | Xie, Ruosen; Wang, Xiuxiu; Wang, Yuyuan; Ye, Mingzhou; Zhao, Yi; Yandell, Brian S; Gong, Shaoqin | Advanced materials (Deerfield Beach, Fla.) | 35119139 | 2022 Feb 04 |
Phage Display-Derived Peptide for the Specific Binding of SARS-CoV-2. | Yang, Fan; Liu, Li; Neuenschwander, Pierre Fernand; Idell, Steven; Vankayalapati, Ramakrishna; Jain, Krishan Gopal; Du, Ke; Ji, Honglong; Yi, Guohua | ACS omega | 35128233 | 2022 Feb 01 |
Species-dependent in vivo mRNA delivery and cellular responses to nanoparticles. | Hatit, Marine Z C; Lokugamage, Melissa P; Dobrowolski, Curtis N; Paunovska, Kalina; Ni, Huanzhen; Zhao, Kun; Vanover, Daryll; Beyersdorf, Jared; Peck, Hannah E; Loughrey, David; Sato, Manaka; Cristian, Ana; Santangelo, Philip J; Dahlman, James E | Nature nanotechnology | 35132167 | 2022 Mar |
A Human-derived Dual MRI/PET Reporter Gene System with High Translational Potential for Cell Tracking. | Shalaby, Nourhan; Kelly, John; Martinez, Francisco; Fox, Mathew; Qi, Qi; Thiessen, Jonathan; Hicks, Justin; Scholl, Timothy J; Ronald, John A | Molecular imaging and biology | 35146614 | 2022 04 |
Nonhuman Primates in Translational Research. | Tarantal, Alice F; Noctor, Stephen C; Hartigan-O'Connor, Dennis J | Annual review of animal biosciences | 35167321 | 2022 02 15 |
A self-immolative linker that releases thiols detects penicillin amidase and nitroreductase with high sensitivity via absorption spectroscopy. | Espinoza, Eli M; Røise, Joachim J; He, Maomao; Li, I-Che; Agatep, Alvin K; Udenyi, Patrick; Han, Hesong; Jackson, Nicole; Kerr, D Lucas; Chen, Dake; Stentzel, Michael R; Ruan, Emily; Riley, Lee; Murthy, Niren | Chemical communications (Cambridge, England) | 35170593 | 2022 Mar 03 |
Intestinal Deletion of 3-Hydroxy-3-Methylglutaryl-Coenzyme A Reductase Promotes Expansion of the Resident Stem Cell Compartment. | Doerfler, Alexandria M; Han, Jun; Jarrett, Kelsey E; Tang, Li; Jain, Antrix; Saltzman, Alexander; De Giorgi, Marco; Chuecos, Marcel; Hurley, Ayrea E; Li, Ang; Morand, Pauline; Ayala, Claudia; Goodlett, David R; Malovannaya, Anna; Martin, James F; de Aguiar Vallim, Thomas Q; Shroyer, Noah; Lagor, William R | Arteriosclerosis, thrombosis, and vascular biology | 35172604 | 2022 Apr |
Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis. | Qiu, Min; Tang, Yan; Chen, Jinjin; Muriph, Rachel; Ye, Zhongfeng; Huang, Changfeng; Evans, Jason; Henske, Elizabeth P; Xu, Qiaobing | Proceedings of the National Academy of Sciences of the United States of America | 35173043 | 2022 02 22 |
Derivation of extra-embryonic and intra-embryonic macrophage lineages from human pluripotent stem cells. | Bredemeyer, Andrea L; Amrute, Junedh M; Koenig, Andrew L; Idol, Rachel A; He, Li; Luff, Stephanie A; Dege, Carissa; Leid, Jamison M; Schilling, Joel D; Hinson, J Travis; Dinauer, Mary C; Sturgeon, Christopher M; Lavine, Kory J | Development (Cambridge, England) | 35178561 | 2022 04 15 |
Transcription factor protein interactomes reveal genetic determinants in heart disease. | Gonzalez-Teran, Barbara; Pittman, Maureen; Felix, Franco; Thomas, Reuben; Richmond-Buccola, Desmond; Hüttenhain, Ruth; Choudhary, Krishna; Moroni, Elisabetta; Costa, Mauro W; Huang, Yu; Padmanabhan, Arun; Alexanian, Michael; Lee, Clara Youngna; Maven, Bonnie E J; Samse-Knapp, Kaitlen; Morton, Sarah U; McGregor, Michael; Gifford, Casey A; Seidman, J G; Seidman, Christine E; Gelb, Bruce D; Colombo, Giorgio; Conklin, Bruce R; Black, Brian L; Bruneau, Benoit G; Krogan, Nevan J; Pollard, Katherine S; Srivastava, Deepak | Cell | 35182466 | 2022 03 03 |
Rapid, accurate mapping of transgene integration in viable rhesus macaque embryos using enhanced-specificity tagmentation-assisted PCR. | Ryu, Junghyun; Chan, William; Wettengel, Jochen M; Hanna, Carol B; Burwitz, Benjamin J; Hennebold, Jon D; Bimber, Benjamin N | Molecular therapy. Methods & clinical development | 35211637 | 2022 Mar 10 |
Magnetic Iron Oxide Nanoparticles for Biomedical Applications. | Jiang, Kaiyi; Zhang, Linlin; Bao, Gang | Current opinion in biomedical engineering | 35211662 | 2021 Dec |
Treating Cardiovascular Disease with Liver Genome Engineering. | Hurley, Ayrea; Lagor, William R | Current atherosclerosis reports | 35230602 | 2022 Feb |
Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challenges. | Schmidt, Jenna K; Jones, Kathryn M; Van Vleck, Trevor; Emborg, Marina E | Science translational medicine | 35235338 | 2022 03 02 |
Modeling injury and repair in kidney organoids reveals that homologous recombination governs tubular intrinsic repair. | Gupta, Navin; Matsumoto, Takuya; Hiratsuka, Ken; Garcia Saiz, Edgar; Galichon, Pierre; Miyoshi, Tomoya; Susa, Koichiro; Tatsumoto, Narihito; Yamashita, Michifumi; Morizane, Ryuji | Science translational medicine | 35235339 | 2022 Mar 02 |
Antispacer peptide nucleic acids for sequence-specific CRISPR-Cas9 modulation. | Economos, Nicholas G; Quijano, Elias; Carufe, Kelly E W; Perera, J Dinithi R; Glazer, Peter M | Nucleic acids research | 35235944 | 2022 Mar 03 |
In Vivo Gene Editing in Lipid and Atherosclerosis Research. | De Giorgi, Marco; Jarrett, Kelsey E; de Aguiar Vallim, Thomas Q; Lagor, William R | Methods in molecular biology (Clifton, N.J.) | 35237996 | 2022 |
Clinical magnetic hyperthermia requires integrated magnetic particle imaging. | Healy, Sean; Bakuzis, Andris F; Goodwill, Patrick W; Attaluri, Anilchandra; Bulte, Jeff W M; Ivkov, Robert | Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology | 35238181 | 2022 May |
Design of therapeutic biomaterials to control inflammation. | Tu, Zhaoxu; Zhong, Yiling; Hu, Hanze; Shao, Dan; Haag, Rainer; Schirner, Michael; Lee, Jaewoo; Sullenger, Bruce; Leong, Kam W | Nature reviews. Materials | 35251702 | 2022 Feb 28 |
Technologies to Assess Drug Response and Heterogeneity in Patient-Derived Cancer Organoids. | Skala, Melissa C; Deming, Dustin A; Kratz, Jeremy D | Annual review of biomedical engineering | 35259932 | 2022 Feb 08 |
Durability of transgene expression after rAAV gene therapy. | Muhuri, Manish; Levy, Daniel I; Schulz, Martin; McCarty, Douglas; Gao, Guangping | Molecular therapy : the journal of the American Society of Gene Therapy | 35283274 | 2022 04 06 |
Translational Utility of the Nonhuman Primate Model. | Tarantal, Alice F; Hartigan-O'Connor, Dennis J; Noctor, Stephen C | Biological psychiatry. Cognitive neuroscience and neuroimaging | 35283343 | 2022 05 |
Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity. | Tran, Ngoc Tam; Lecomte, Emilie; Saleun, Sylvie; Namkung, Suk; Robin, Cécile; Weber, Kristina; Devine, Eric; Blouin, Veronique; Adjali, Oumeya; Ayuso, Eduard; Gao, Guangping; Penaud-Budloo, Magalie; Tai, Phillip W L | Human gene therapy | 35293222 | 2022 04 |
Toward improved understanding of cardiac development and congenital heart disease: The advent of cardiac organoids. | Scherba, Jacob C; Karra, Ravi; Turek, Joseph W; Bursac, Nenad | The Journal of thoracic and cardiovascular surgery | 35307217 | 2022 Dec |
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice. | Wang, Jiaming; Zhang, Yue; Mendonca, Craig A; Yukselen, Onur; Muneeruddin, Khaja; Ren, Lingzhi; Liang, Jialing; Zhou, Chen; Xie, Jun; Li, Jia; Jiang, Zhong; Kucukural, Alper; Shaffer, Scott A; Gao, Guangping; Wang, Dan | Nature | 35322228 | 2022 04 |
Bioengineered Kidney Models: Methods and Functional Assessments. | Rizki-Safitri, Astia; Traitteur, Tamara; Morizane, Ryuji | Function (Oxford, England) | 35330622 | 2021 |
Impact of baseline culture conditions of cancer organoids when determining therapeutic response and tumor heterogeneity. | DeStefanis, Rebecca A; Kratz, Jeremy D; Olson, Autumn M; Sunil, Aishwarya; DeZeeuw, Alyssa K; Gillette, Amani A; Sha, Gioia C; Johnson, Katherine A; Pasch, Cheri A; Clipson, Linda; Skala, Melissa C; Deming, Dustin A | Scientific reports | 35338174 | 2022 03 25 |
Robust, Durable Gene Activation In Vivo via mRNA-Encoded Activators. | Beyersdorf, Jared P; Bawage, Swapnil; Iglesias, Nahid; Peck, Hannah E; Hobbs, Ryan A; Wroe, Jay A; Zurla, Chiara; Gersbach, Charles A; Santangelo, Philip J | ACS nano | 35357116 | 2022 Mar 31 |
Efficient targeted transgenesis of large donor DNA into multiple mouse genetic backgrounds using bacteriophage Bxb1 integrase. | Low, Benjamin E; Hosur, Vishnu; Lesbirel, Simon; Wiles, Michael V | Scientific reports | 35361849 | 2022 03 31 |
Physiology assays in human kidney organoids. | Freedman, Benjamin S | American journal of physiology. Renal physiology | 35379001 | 2022 06 01 |
CRISPR-free base editors with enhanced activity and expanded targeting scope in mitochondrial and nuclear DNA. | Mok, Beverly Y; Kotrys, Anna V; Raguram, Aditya; Huang, Tony P; Mootha, Vamsi K; Liu, David R | Nature biotechnology | 35379961 | 2022 Apr 04 |
A split prime editor with untethered reverse transcriptase and circular RNA template. | Liu, Bin; Dong, Xiaolong; Cheng, Haoyang; Zheng, Chunwei; Chen, Zexiang; Rodríguez, Tomás C; Liang, Shun-Qing; Xue, Wen; Sontheimer, Erik J | Nature biotechnology | 35379962 | 2022 09 |
In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration. | Choi, Elliot H; Suh, Susie; Foik, Andrzej T; Leinonen, Henri; Newby, Gregory A; Gao, Xin D; Banskota, Samagya; Hoang, Thanh; Du, Samuel W; Dong, Zhiqian; Raguram, Aditya; Kohli, Sajeev; Blackshaw, Seth; Lyon, David C; Liu, David R; Palczewski, Krzysztof | Nature communications | 35383196 | 2022 Apr 05 |
Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells. | Luks, Valerie L; Mandl, Hanna; DiRito, Jenna; Barone, Christina; Freedman-Weiss, Mollie R; Ricciardi, Adele S; Tietjen, Gregory G; Egan, Marie E; Saltzman, W Mark; Stitelman, David H | PloS one | 35385514 | 2022 |
Multivalent designed proteins neutralize SARS-CoV-2 variants of concern and confer protection against infection in mice. | Hunt, Andrew C; Case, James Brett; Park, Young-Jun; Cao, Longxing; Wu, Kejia; Walls, Alexandra C; Liu, Zhuoming; Bowen, John E; Yeh, Hsien-Wei; Saini, Shally; Helms, Louisa; Zhao, Yan Ting; Hsiang, Tien-Ying; Starr, Tyler N; Goreshnik, Inna; Kozodoy, Lisa; Carter, Lauren; Ravichandran, Rashmi; Green, Lydia B; Matochko, Wadim L; Thomson, Christy A; Vögeli, Bastian; Krüger, Antje; VanBlargan, Laura A; Chen, Rita E; Ying, Baoling; Bailey, Adam L; Kafai, Natasha M; Boyken, Scott E; Ljubetič, Ajasja; Edman, Natasha; Ueda, George; Chow, Cameron M; Johnson, Max; Addetia, Amin; Navarro, Mary Jane; Panpradist, Nuttada; Gale Jr, Michael; Freedman, Benjamin S; Bloom, Jesse D; Ruohola-Baker, Hannele; Whelan, Sean P J; Stewart, Lance; Diamond, Michael S; Veesler, David; Jewett, Michael C; Baker, David | Science translational medicine | 35412328 | 2022 Apr 12 |
Myoblast deactivation within engineered human skeletal muscle creates a transcriptionally heterogeneous population of quiescent satellite-like cells. | Wang, Jason; Broer, Torie; Chavez, Taylor; Zhou, Chris J; Tran, Sabrina; Xiang, Yu; Khodabukus, Alastair; Diao, Yarui; Bursac, Nenad | Biomaterials | 35421801 | 2022 05 |
CRISPR-Cas9 bends and twists DNA to read its sequence. | Cofsky, Joshua C; Soczek, Katarzyna M; Knott, Gavin J; Nogales, Eva; Doudna, Jennifer A | Nature structural & molecular biology | 35422516 | 2022 Apr |
Crystal structure of an RNA/DNA strand exchange junction. | Cofsky, Joshua C; Knott, Gavin J; Gee, Christine L; Doudna, Jennifer A | PloS one | 35436289 | 2022 |
Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome Editing. | Chen, Zexiang; Devi, Gitali; Arif, Amena; Zamore, Phillip D; Sontheimer, Erik J; Watts, Jonathan K | ACS chemical biology | 35446558 | 2022 05 20 |
Modelling ciliopathy phenotypes in human tissues derived from pluripotent stem cells with genetically ablated cilia. | Cruz, Nelly M; Reddy, Raghava; McFaline-Figueroa, José L; Tran, Christine; Fu, Hongxia; Freedman, Benjamin S | Nature biomedical engineering | 35478224 | 2022 04 |
An optimized FusX assembly-based technique to introduce mitochondrial TC-to-TT variations in human cell lines. | Kar, Bibekananda; Sabharwal, Ankit; Restrepo-Castillo, Santiago; Simone, Brandon W; Clark, Karl J; Ekker, Stephen C | STAR protocols | 35496789 | 2022 Jun 17 |
Identification and Validation of CRISPR/Cas9 Off-Target Activity in Hematopoietic Stem and Progenitor Cells. | Park, So Hyun; Lee, Ciaran M; Bao, Gang | Methods in molecular biology (Clifton, N.J.) | 35507169 | 2022 |
Prioritization of autoimmune disease-associated genetic variants that perturb regulatory element activity in T cells. | Mouri, Kousuke; Guo, Michael H; de Boer, Carl G; Lissner, Michelle M; Harten, Ingrid A; Newby, Gregory A; DeBerg, Hannah A; Platt, Winona F; Gentili, Matteo; Liu, David R; Campbell, Daniel J; Hacohen, Nir; Tewhey, Ryan; Ray, John P | Nature genetics | 35513721 | 2022 05 |
The clinical progress of mRNA vaccines and immunotherapies. | Barbier, Ann J; Jiang, Allen Yujie; Zhang, Peng; Wooster, Richard; Anderson, Daniel G | Nature biotechnology | 35534554 | 2022 06 |
Structural biology of CRISPR-Cas immunity and genome editing enzymes. | Wang, Joy Y; Pausch, Patrick; Doudna, Jennifer A | Nature reviews. Microbiology | 35562427 | 2022 11 |
A High-Efficiency AAV for Endothelial Cell Transduction Throughout the Central Nervous System. | Krolak, Trevor; Chan, Ken Y; Kaplan, Luke; Huang, Qin; Wu, Jason; Zheng, Qingxia; Kozareva, Velina; Beddow, Thomas; Tobey, Isabelle G; Pacouret, Simon; Chen, Albert T; Chan, Yujia A; Ryvkin, Daniel; Gu, Chenghua; Deverman, Benjamin E | Nature cardiovascular research | 35571675 | 2022 Apr |
Label-Free Imaging to Track Reprogramming of Human Somatic Cells. | Molugu, Kaivalya; Battistini, Giovanni A; Heaster, Tiffany M; Rouw, Jacob; Guzman, Emmanuel C; Skala, Melissa C; Saha, Krishanu | GEN biotechnology | 35586336 | 2022 Apr 01 |
CRISPR Library Screening in Cultured Cardiomyocytes. | DeLuca, Sophia; Bursac, Nenad | Methods in molecular biology (Clifton, N.J.) | 35618895 | 2022 |
Imaging the fetal nonhuman primate brain with SV2A positron emission tomography (PET). | Rossano, Samantha; Toyonaga, Takuya; Berg, Eric; Lorence, Isabella; Fowles, Krista; Nabulsi, Nabeel; Ropchan, Jim; Li, Songye; Ye, Yunpeng; Felchner, Zachary; Kukis, David; Huang, Yiyun; Benveniste, Helene; Tarantal, Alice F; Groman, Stephanie; Carson, Richard E | European journal of nuclear medicine and molecular imaging | 35633376 | 2022 09 |
Inhibition of B-cell lymphoma 2 family proteins alters optical redox ratio, mitochondrial polarization, and cell energetics independent of cell state. | Gillette, Amani A; DeStefanis, Rebecca A; Pritzl, Stephanie L; Deming, Dustin A; Skala, Melissa C | Journal of biomedical optics | 35643815 | 2022 05 |
Synthesis and ex vivo biodistribution of two 68Ga-labeled tetrazine tracers: Comparison of pharmacokinetics. | Lambidis, Elisavet; Lumén, Dave; Koskipahta, Elina; Imlimthan, Surachet; Lopez, Brianda B; Sánchez, Ana Isabel Fraguas; Sarparanta, Mirkka; Cheng, R Holland; Airaksinen, Anu J | Nuclear medicine and biology | 35680503 | 2022 |
In vivo engineered B cells secrete high titers of broadly neutralizing anti-HIV antibodies in mice. | Nahmad, Alessio D; Lazzarotto, Cicera R; Zelikson, Natalie; Kustin, Talia; Tenuta, Mary; Huang, Deli; Reuveni, Inbal; Nataf, Daniel; Raviv, Yuval; Horovitz-Fried, Miriam; Dotan, Iris; Carmi, Yaron; Rosin-Arbesfeld, Rina; Nemazee, David; Voss, James E; Ste | Nature biotechnology | 35681059 | 2022 Aug |
The balance between protective and pathogenic immune responses to pneumonia in the neonatal lung is enforced by gut microbiota. | Stevens, Joseph; Steinmeyer, Shelby; Bonfield, Madeline; Peterson, Laura; Wang, Timothy; Gray, Jerilyn; Lewkowich, Ian; Xu, Yan; Du, Yina; Guo, Minzhe; Wynn, James L; Zacharias, William; Salomonis, Nathan; Miller, Lisa; Chougnet, Claire; O'Connor, Dennis Hartigan; Deshmukh, Hitesh | Science translational medicine | 35704600 | 2022 Jun 15 |
Efficient Genome Editing Achieved via Plug-and-Play Adenovirus Piggyback Transport of Cas9/gRNA Complex on Viral Capsid Surface. | Lu, Zhi Hong; Li, Jie; Dmitriev, Igor P; Kashentseva, Elena A; Curiel, David T | ACS nano | 35749339 | 2022 Jun 24 |
Kidney organoids: a pioneering model for kidney diseases. | Tekguc, Murat; Gaal, Ronald C VAN; Uzel, Sebastien G M; Gupta, Navin; Riella, Leonardo V; Lewis, Jennifer A; Morizane, Ryuji | Translational research : the journal of laboratory and clinical medicine | 35750295 | 2022 Jun 21 |
Nanoparticle single-cell multiomic readouts reveal that cell heterogeneity influences lipid nanoparticle-mediated messenger RNA delivery. | Dobrowolski, Curtis; Paunovska, Kalina; Schrader Echeverri, Elisa; Loughrey, David; Da Silva Sanchez, Alejandro J; Ni, Huanzhen; Hatit, Marine Z C; Lokugamage, Melissa P; Kuzminich, Yanina; Peck, Hannah E; Santangelo, Philip J; Dahlman, James E | Nature nanotechnology | 35768613 | 2022 08 |
In situ autofluorescence lifetime assay of a photoreceptor stimulus response in mouse retina and human retinal organoids. | Samimi, Kayvan; Pattnaik, Bikash R; Capowski, Elizabeth E; Saha, Krishanu; Gamm, David M; Skala, Melissa C | Biomedical optics express | 35781966 | 2022 Jun 01 |
Therapeutic in vivo delivery of gene editing agents. | Raguram, Aditya; Banskota, Samagya; Liu, David R | Cell | 35798006 | 2022 Jul 21 |
SARS-CoV-2 Host Immunogenetic Biomarkers. | Gededzha, Maemu P; Mampeule, Nakampe; Gandini, Anastasia; Mayne, Elizabeth S | Methods in molecular biology (Clifton, N.J.) | 35838957 | 2022 |
Evaluation Protocol for SARS-CoV-2 Serological Assays. | Gededzha, Maemu P; Jugwanth, Sarika; Mampeule, Nakampe; Zwane, Nontobeko; David, Anura; Scott, Lesley; Stevens, Wendy; Mayne, Elizabeth S | Methods in molecular biology (Clifton, N.J.) | 35838970 | 2022 |
Non-Invasive imaging of extracellular vesicles: Quo vaditis in vivo? | Arifin, Dian R; Witwer, Kenneth W; Bulte, Jeff W M | Journal of extracellular vesicles | 35844061 | 2022 Jul |
Development of 68Ga-Labeled Hepatitis E Virus Nanoparticles for Targeted Drug Delivery and Diagnostics with PET. | Lambidis, Elisavet; Chen, Chun-Chieh; Baikoghli, Mo; Imlimthan, Surachet; Khng, You Cheng; Sarparanta, Mirkka; Cheng, R Holland; Airaksinen, Anu J | Molecular pharmaceutics | 35857429 | 2022 08 01 |
Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors. | Davis, Jessie R; Wang, Xiao; Witte, Isaac P; Huang, Tony P; Levy, Jonathan M; Raguram, Aditya; Banskota, Samagya; Seidah, Nabil G; Musunuru, Kiran; Liu, David R | Nature biomedical engineering | 35902773 | 2022 Jul 28 |
Multiomics characterization of mouse hepatoblastoma identifies yes-associated protein 1 target genes. | Rodríguez, Tomás C; Kwan, Suet-Yan; Smith, Jordan L; Dadafarin, Sina; Wu, Chern-Horng; Sontheimer, Erik J; Xue, Wen | Hepatology (Baltimore, Md.) | 35932276 | 2022 Aug 06 |
Designing and executing prime editing experiments in mammalian cells. | Doman, Jordan L; Sousa, Alexander A; Randolph, Peyton B; Chen, Peter J; Liu, David R | Nature protocols | 35941224 | 2022 Aug 08 |
Material Engineering in Gut Microbiome and Human Health. | Yang, Letao; Hung, Lin Y; Zhu, Yuefei; Ding, Suwan; Margolis, Kara G; Leong, Kam W | Research (Washington, D.C.) | 35958108 | 2022 |
Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivo. | Ni, Huanzhen; Hatit, Marine Z C; Zhao, Kun; Loughrey, David; Lokugamage, Melissa P; Peck, Hannah E; Cid, Ada Del; Muralidharan, Abinaya; Kim, YongTae; Santangelo, Philip J; Dahlman, James E | Nature communications | 35970837 | 2022 08 15 |
Prostate cancer cells demonstrate unique metabolism and substrate adaptability acutely after androgen deprivation therapy. | Filon, Mikolaj J; Gillette, Amani A; Yang, Bing; Khemees, Tariq A; Skala, Melissa C; Jarrard, David F | The Prostate | 35980831 | 2022 Dec |
An ELISA-based platform for rapid identification of structure-dependent nucleic acid-protein interactions detects novel DNA triplex interactors. | Economos, Nicholas G; Thapar, Upasna; Balasubramanian, Nanda; Karras, Georgios I; Glazer, Peter M | The Journal of biological chemistry | 35988651 | 2022 Oct |
High-throughput continuous evolution of compact Cas9 variants targeting single-nucleotide-pyrimidine PAMs. | Huang, Tony P; Heins, Zachary J; Miller, Shannon M; Wong, Brandon G; Balivada, Pallavi A; Wang, Tina; Khalil, Ahmad S; Liu, David R | Nature biotechnology | 36076084 | 2022 Sep 08 |
Differential microRNA profiles of intramuscular and secreted extracellular vesicles in human tissue-engineered muscle. | Vann, Christopher G; Zhang, Xin; Khodabukus, Alastair; Orenduff, Melissa C; Chen, Yu-Hsiu; Corcoran, David L; Truskey, George A; Bursac, Nenad; Kraus, Virginia B | Frontiers in physiology | 36091396 | 2022 |
Wnt5a/β-catenin axis is involved in the downregulation of AT2 lineage by PAI-1. | Jain, Krishan G; Zhao, Runzhen; Liu, Yang; Guo, Xuan; Yi, Guohua; Ji, Hong-Long | American journal of physiology. Lung cellular and molecular physiology | 36098461 | 2022 Nov 01 |
Precision genome editing in the eye. | Suh, Susie; Choi, Elliot H; Raguram, Aditya; Liu, David R; Palczewski, Krzysztof | Proceedings of the National Academy of Sciences of the United States of America | 36122230 | 2022 Sep 27 |
Promoting validation and cross-phylogenetic integration in model organism research. | Cheng, Keith C; Burdine, Rebecca D; Dickinson, Mary E; Ekker, Stephen C; Lin, Alex Y; Lloyd, K C Kent; Lutz, Cathleen M; MacRae, Calum A; Morrison, John H; O'Connor, David H; Postlethwait, John H; Rogers, Crystal D; Sanchez, Susan; Simpson, Julie H; Talbot, William S; Wallace, Douglas C; Weimer, Jill M; Bellen, Hugo J | Disease models & mechanisms | 36125045 | 2022 09 01 |
Organoid-on-a-chip model of human ARPKD reveals mechanosensing pathomechanisms for drug discovery. | Hiratsuka, Ken; Miyoshi, Tomoya; Kroll, Katharina T; Gupta, Navin R; Valerius, M Todd; Ferrante, Thomas; Yamashita, Michifumi; Lewis, Jennifer A; Morizane, Ryuji | Science advances | 36129975 | 2022 09 23 |
Engineering multicellular living systems-A Keystone Symposia report. | Cable, Jennifer; Arlotta, Paola; Parker, Kevin Kit; Hughes, Alex J; Goodwin, Katharine; Mummery, Christine L; Kamm, Roger D; Engle, Sandra J; Tagle, Danilo A; Boj, Sylvia F; Stanton, Alice E; Morishita, Yoshihiro; Kemp, Melissa L; Norfleet, Dennis A; May, | Annals of the New York Academy of Sciences | 36177947 | 2022 Sep 30 |
Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes. | Namkung, Suk; Tran, Ngoc Tam; Manokaran, Sangeetha; He, Ran; Su, Qin; Xie, Jun; Gao, Guangping; Tai, Phillip W L | Human gene therapy | 36178359 | 2022 Nov |
In vivo correction of cystic fibrosis mediated by PNA nanoparticles. | Piotrowski-Daspit, Alexandra S; Barone, Christina; Lin, Chun-Yu; Deng, Yanxiang; Wu, Douglas; Binns, Thomas C; Xu, Emily; Ricciardi, Adele S; Putman, Rachael; Garrison, Alannah; Nguyen, Richard; Gupta, Anisha; Fan, Rong; Glazer, Peter M; Saltzman, W Mark; Egan, Marie E | Science advances | 36197984 | 2022 Oct 07 |
Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. | Gonzalez, Trevor J; Simon, Katherine E; Blondel, Leo O; Fanous, Marco M; Roger, Angela L; Maysonet, Maribel Santiago; Devlin, Garth W; Smith, Timothy J; Oh, Daniel K; Havlik, L Patrick; Castellanos Rivera, Ruth M; Piedrahita, Jorge A; ElMallah, Mai K; Ger | Nature communications | 36210364 | 2022 Oct 10 |
EZ Clear for simple, rapid, and robust mouse whole organ clearing. | Hsu, Chih-Wei; Cerda, Juan; Kirk, Jason M; Turner, Williamson D; Rasmussen, Tara L; Flores Suarez, Carlos P; Dickinson, Mary E; Wythe, Joshua D | eLife | 36218247 | 2022 Oct 11 |
Monobody adapter for functional antibody display on nanoparticles for adaptable targeted delivery applications. | Albert, C; Bracaglia, L; Koide, A; DiRito, J; Lysyy, T; Harkins, L; Edwards, C; Richfield, O; Grundler, J; Zhou, K; Denbaum, E; Ketavarapu, G; Hattori, T; Perincheri, S; Langford, J; Feizi, A; Haakinson, D; Hosgood, S A; Nicholson, M L; Pober, J S; Saltzman, W M; Koide, S; Tietjen, G T | Nature communications | 36220817 | 2022 Oct 11 |
Translating musculoskeletal bioengineering into tissue regeneration therapies. | Khodabukus, Alastair; Guyer, Tyler; Moore, Axel C; Stevens, Molly M; Guldberg, Robert E; Bursac, Nenad | Science translational medicine | 36223445 | 2022 Oct 12 |
Nucleic Acid Delivery to the Vascular Endothelium. | Reschke, Melanie; Piotrowski-Daspit, Alexandra S; Pober, Jordan S; Saltzman, W Mark | Molecular pharmaceutics | 36251765 | 2022 Dec 05 |
Unadjuvanted intranasal spike vaccine elicits protective mucosal immunity against sarbecoviruses. | Mao, Tianyang; Israelow, Benjamin; Peña-Hernández, Mario A; Suberi, Alexandra; Zhou, Liqun; Luyten, Sophia; Reschke, Melanie; Dong, Huiping; Homer, Robert J; Saltzman, W Mark; Iwasaki, Akiko | Science (New York, N.Y.) | 36302057 | 2022 Nov 25 |
Adult human kidney organoids originate from CD24+ cells and represent an advanced model for adult polycystic kidney disease. | Xu, Yaoxian; Kuppe, Christoph; Perales-Patón, Javier; Hayat, Sikander; Kranz, Jennifer; Abdallah, Ali T; Nagai, James; Li, Zhijian; Peisker, Fabian; Saritas, Turgay; Halder, Maurice; Menzel, Sylvia; Hoeft, Konrad; Kenter, Annegien; Kim, Hyojin; van Roeyen, Claudia R C; Lehrke, Michael; Moellmann, Julia; Speer, Thimoteus; Buhl, Eva M; Hoogenboezem, Remco; Boor, Peter; Jansen, Jitske; Knopp, Cordula; Kurth, Ingo; Smeets, Bart; Bindels, Eric; Reinders, Marlies E J; Baan, Carla; Gribnau, Joost; Hoorn, Ewout J; Steffens, Joachim; Huber, Tobias B; Costa, Ivan; Floege, Jürgen; Schneider, Rebekka K; Saez-Rodriguez, Julio; Freedman, Benjamin S; Kramann, Rafael | Nature genetics | 36303074 | 2022 Oct 27 |
Prime editing for precise and highly versatile genome manipulation. | Chen, Peter J; Liu, David R | Nature reviews. Genetics | 36344749 | 2023 Mar |
Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity. | Neugebauer, Monica E; Hsu, Alvin; Arbab, Mandana; Krasnow, Nicholas A; McElroy, Amber N; Pandey, Smriti; Doman, Jordan L; Huang, Tony P; Raguram, Aditya; Banskota, Samagya; Newby, Gregory A; Tolar, Jakub; Osborn, Mark J; Liu, David R | Nature biotechnology | 36357719 | 2022 Nov 10 |
LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study. | Doerfler, Alexandria M; Park, So Hyun; Assini, Julia M; Youssef, Amer; Saxena, Lavanya; Yaseen, Adam B; De Giorgi, Marco; Chuecos, Marcel; Hurley, Ayrea E; Li, Ang; Marcovina, Santica M; Bao, Gang; Boffa, Michael B; Koschinsky, Marlys L; Lagor, William R | Molecular therapy. Methods & clinical development | 36381302 | 2022 Dec 08 |
Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression. | Mueller, Katherine P; Piscopo, Nicole J; Forsberg, Matthew H; Saraspe, Louise A; Das, Amritava; Russell, Brittany; Smerchansky, Madeline; Cappabianca, Dan; Shi, Lei; Shankar, Keerthana; Sarko, Lauren; Khajanchi, Namita; La Vonne Denne, Nina; Ramamurthy, A | Journal for immunotherapy of cancer | 36382633 | 2022 Sep |
ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation. | Sheriff, Adam; Guri, Ina; Zebrowska, Paulina; Llopis-Hernandez, Virginia; Brooks, Imogen R; Tekkela, Stavroula; Subramaniam, Kavita; Gebrezgabher, Ruta; Naso, Gaetano; Petrova, Anastasia; Balon, Katarzyna; Onoufriadis, Alexandros; Kujawa, Dorota; Kotulska, Martyna; Newby, Gregory; Łaczmański, Łukasz; Liu, David R; McGrath, John A; Jacków, Joanna | Scientific reports | 36385635 | 2022 Nov 16 |
Compact zinc finger base editors that edit mitochondrial or nuclear DNA in vitro and in vivo. | Willis, Julian C W; Silva-Pinheiro, Pedro; Widdup, Lily; Minczuk, Michal; Liu, David R | Nature communications | 36418298 | 2022 Nov 23 |
Diverse virus-encoded CRISPR-Cas systems include streamlined genome editors. | Al-Shayeb, Basem; Skopintsev, Petr; Soczek, Katarzyna M; Stahl, Elizabeth C; Li, Zheng; Groover, Evan; Smock, Dylan; Eggers, Amy R; Pausch, Patrick; Cress, Brady F; Huang, Carolyn J; Staskawicz, Brian; Savage, David F; Jacobsen, Steven E; Banfield, Jillia | Cell | 36423580 | 2022 Nov 23 |
Substituting racemic ionizable lipids with stereopure ionizable lipids can increase mRNA delivery. | Da Silva Sanchez, Alejandro J; Zhao, Kun; Huayamares, Sebastian G; Hatit, Marine Z C; Lokugamage, Melissa P; Loughrey, David; Dobrowolski, Curtis; Wang, Shuaishuai; Kim, Hyejin; Paunovska, Kalina; Kuzminich, Yanina; Dahlman, James E | Journal of controlled release : official journal of the Controlled Release Society | 36423872 | 2022 Nov 30 |
Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases. | Yarnall, Matthew T N; Ioannidi, Eleonora I; Schmitt-Ulms, Cian; Krajeski, Rohan N; Lim, Justin; Villiger, Lukas; Zhou, Wenyuan; Jiang, Kaiyi; Garushyants, Sofya K; Roberts, Nathaniel; Zhang, Liyang; Vakulskas, Christopher A; Walker 2nd, John A; Kadina, Anastasia P; Zepeda, Adrianna E; Holden, Kevin; Ma, Hong; Xie, Jun; Gao, Guangping; Foquet, Lander; Bial, Greg; Donnelly, Sara K; Miyata, Yoshinari; Radiloff, Daniel R; Henderson, Jordana M; Ujita, Andrew; Abudayyeh, Omar O; Gootenberg, Jonathan S | Nature biotechnology | 36424489 | 2023 Apr |
Cyclin G1 induces maladaptive proximal tubule cell dedifferentiation and renal fibrosis through CDK5 activation. | Taguchi, Kensei; Elias, Bertha C; Sugahara, Sho; Sant, Snehal; Freedman, Benjamin S; Waikar, Sushrut S; Pozzi, Ambra; Zent, Roy; Harris, Raymond C; Parikh, Samir M; Brooks, Craig R | The Journal of clinical investigation | 36453545 | 2022 Dec 01 |
Reframing the perception of outliers and negative data in translational research. | Emborg, Marina E | Brain research bulletin | 36464129 | 2023 Jan |
An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair. | Yan, Ruorong; Cigliola, Valentina; Oonk, Kelsey A; Petrover, Zachary; DeLuca, Sophia; Wolfson, David W; Vekstein, Andrew; Mendiola, Michelle A; Devlin, Garth; Bishawi, Muath; Gemberling, Matthew P; Sinha, Tanvi; Sargent, Michelle A; York, Allen J; Shakked, Avraham; DeBenedittis, Paige; Wendell, David C; Ou, Jianhong; Kang, Junsu; Goldman, Joseph A; Baht, Gurpreet S; Karra, Ravi; Williams, Adam R; Bowles, Dawn E; Asokan, Aravind; Tzahor, Eldad; Gersbach, Charles A; Molkentin, Jeffery D; Bursac, Nenad; Black, Brian L; Poss, Kenneth D | Cell stem cell | 36516837 | 2023 Jan 05 |
Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteins. | Metzger, Jeanette M; Wang, Yuyuan; Neuman, Samuel S; Snow, Kathy J; Murray, Stephen A; Lutz, Cathleen M; Bondarenko, Viktoriya; Felton, Jesi; Gimse, Kirstan; Xie, Ruosen; Li, Dongdong; Zhao, Yi; Flowers, Matthew T; Simmons, Heather A; Roy, Subhojit; Saha, Krishanu; Levine, Jon E; Emborg, Marina E; Gong, Shaoqin | Biomaterials | 36527789 | 2022 Dec 13 |
Emerging immunomodulatory strategies for cell therapeutics. | Chua, Corrine Ying Xuan; Jiang, Allen Yujie; Eufrásio-da-Silva, Tatiane; Dolatshahi-Pirouz, Alireza; Langer, Robert; Orive, Gorka; Grattoni, Alessandro | Trends in biotechnology | 36549959 | 2023 Mar |
Glucose absorption drives cystogenesis in a human organoid-on-chip model of polycystic kidney disease. | Li, Sienna R; Gulieva, Ramila E; Helms, Louisa; Cruz, Nelly M; Vincent, Thomas; Fu, Hongxia; Himmelfarb, Jonathan; Freedman, Benjamin S | Nature communications | 36564419 | 2022 Dec 23 |
Rigid respiration: fulminant pulmonary fibrosis after COVID-19. | Meyerholz, David K | EBioMedicine | 36580850 | 2023 Jan |
In vivo editing of the pan-endothelium by immunity evading simian adenoviral vector. | Lorincz, Reka; Alvarez, Aluet Borrego; Walkey, Christopher J; Mendonça, Samir A; Lu, Zhi Hong; Martinez, Alexa E; Ljungberg, Cecilia; Heaney, Jason D; Lagor, William R; Curiel, David T | Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie | 36587560 | 2023 Feb |
In Vivo Cellular Magnetic Imaging: Labeled vs. Unlabeled Cells. | Bulte, Jeff W M; Wang, Chao; Shakeri-Zadeh, Ali | Advanced functional materials | 36589903 | 2022 Dec 09 |
Accounting for diversity in the design of CRISPR-based therapeutic genome editing. | Saha, Krishanu | Nature genetics | 36593304 | 2023 Jan |
Synthetic Biology Design as a Paradigm Shift toward Manufacturing Affordable Adeno-Associated Virus Gene Therapies. | Collins, Logan Thrasher; Ponnazhagan, Selvarangan; Curiel, David T | ACS synthetic biology | 36627108 | 2023 Jan 20 |
Phenylboronic Acid-Functionalized Polyplexes Tailored to Oral CRISPR Delivery. | Yoshinaga, Naoto; Zhou, Joyce K; Xu, Cong; Quek, Chai Hoon; Zhu, Yuefei; Tang, Ding; Hung, Lin Yung; Najjar, Sarah A; Shiu, Chin Ying Angela; Margolis, Kara Gross; Lao, Yeh-Hsing; Leong, Kam W | Nano letters | 36648291 | 2023 Feb 08 |
Clinical progress in genome-editing technology and in vivo delivery techniques. | Khirallah, Jennifer; Eimbinder, Maximilan; Li, Yamin; Xu, Qiaobing | Trends in genetics : TIG | 36669950 | 2023 Jan 18 |
CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells. | Brault, Julie; Liu, Taylor; Liu, Siyuan; Lawson, Amanda; Choi, Uimook; Kozhushko, Nikita; Bzhilyanskaya, Vera; Pavel-Dinu, Mara; Meis, Ronald J; Eckhaus, Michael A; Burkett, Sandra S; Bosticardo, Marita; Kleinstiver, Benjamin P; Notarangelo, Luigi D; Lazzarotto, Cicera R; Tsai, Shengdar Q; Wu, Xiaolin; Dahl, Gary A; Porteus, Matthew H; Malech, Harry L; De Ravin, Suk See | Frontiers in immunology | 36685559 | 2022 |
Precise transcript targeting by CRISPR-Csm complexes. | Colognori, David; Trinidad, Marena; Doudna, Jennifer A | Nature biotechnology | 36690762 | 2023 Sep |
The Transcriptional Response to Lung-Targeting Lipid Nanoparticles in Vivo. | Radmand, Afsane; Lokugamage, Melissa P; Kim, Hyejin; Dobrowolski, Curtis; Zenhausern, Ryan; Loughrey, David; Huayamares, Sebastian G; Hatit, Marine Z C; Ni, Huanzhen; Del Cid, Ada; Da Silva Sanchez, Alejandro J; Paunovska, Kalina; Schrader Echeverri, Elisa; Shajii, Aram; Peck, Hannah; Santangelo, Philip J; Dahlman, James E | Nano letters | 36701517 | 2023 Feb 08 |
Base editing as a genetic treatment for spinal muscular atrophy. | Alves, Christiano R R; Ha, Leillani L; Yaworski, Rebecca; Lazzarotto, Cicera R; Christie, Kathleen A; Reilly, Aoife; Beauvais, Ariane; Doll, Roman M; de la Cruz, Demitri; Maguire, Casey A; Swoboda, Kathryn J; Tsai, Shengdar Q; Kothary, Rashmi; Kleinstiver, Benjamin P | bioRxiv : the preprint server for biology | 36711797 | 2023 Jan 21 |
Focused ultrasound-mediated brain genome editing. | Leong, Kam; Lao, Yeh-Hsing; Ji, Robin; Zhou, Joyce; Snow, Kathy; Kwon, Nancy; Saville, Ethan; He, Siyu; Chauhan, Shradha; Chi, Chun-Wei; Datta, Malika; Zhang, Hairong; Quek, Chai Hoon; Cai, Sarah; Li, Mingqiang; Gaitan, Yaned; Bechtel, Lawrence; Wu, Shih-Ying; Lutz, Cathleen; Tomer, Raju; Murray, Stephen; Chavez, Alejandro; Konofagou, Elisa | Research square | 36712096 | 2023 Jan 20 |
Functional maturation of kidney organoid tubules: Piezo1-mediated Ca2+ signaling. | Carrisoza-Gaytán, Rolando; Kroll, Katharina T; Hiratsuka, Ken; Gupta, Navin R; Morizane, Ryuji; Lewis, Jennifer A; Satlin, Lisa M | American journal of physiology. Cell physiology | 36745528 | 2023 Feb 06 |
Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice. | Reichart, Daniel; Newby, Gregory A; Wakimoto, Hiroko; Lun, Mingyue; Gorham, Joshua M; Curran, Justin J; Raguram, Aditya; DeLaughter, Daniel M; Conner, David A; Marsiglia, Júlia D C; Kohli, Sajeev; Chmatal, Lukas; Page, David C; Zabaleta, Nerea; Vandenberghe, Luk; Liu, David R; Seidman, Jonathan G; Seidman, Christine | Nature medicine | 36797483 | 2023 Feb |
In vivo HSC prime editing rescues sickle cell disease in a mouse model. | Li, Chang; Georgakopoulou, Aphrodite; Newby, Gregory A; Chen, Peter J; Everette, Kelcee A; Paschoudi, Kiriaki; Vlachaki, Efthymia; Gil, Sucheol; Anderson, Anna K; Koob, Theodore; Huang, Lishan; Wang, Hongjie; Kiem, Hans-Peter; Liu, David R; Yannaki, Evangelia; Lieber, André | Blood | 36800642 | 2023 Apr 27 |
Shuttle Peptide Delivers Base Editor RNPs to Rhesus Monkey Airway Epithelial Cells In Vivo. | Kulhankova, Katarina; Traore, Soumba; Cheng, Xue; Benk-Fortin, Hadrien; Hallée, Stéphanie; Harvey, Mario; Roberge, Joannie; Couture, Frédéric; Gross, Thomas; Newby, Gregory; Liu, David; Tarantal, Alice; Guay, David; McCray, Paul | Research square | 36824928 | 2023 Feb 17 |
In utero delivery of mRNA to the heart, diaphragm and muscle with lipid nanoparticles. | Gao, Kewa; Li, Jie; Song, Hengyue; Han, Hesong; Wang, Yongheng; Yin, Boyan; Farmer, Diana L; Murthy, Niren; Wang, Aijun | Bioactive materials | 36844366 | 2023 Jul |
A parathyroid hormone/salt-inducible kinase signaling axis controls renal vitamin D activation and organismal calcium homeostasis. | Yoon, Sung-Hee; Meyer, Mark B; Arevalo Rivas, Carlos; Tekguc, Murat; Zhang, Chengcheng; Wang, Jialiang S; Castro Andrade, Christian D; Strauss, Katelyn E; Sato, Tadatoshi; Benkusky, Nancy; Lee, Seong Min; Berdeaux, Rebecca; Foretz, Marc; Sundberg, Thomas B; Xavier, Ramnik J; Adelmann, Charles H; Brooks, Daniel J; Anselmo, Anthony; Sadreyev, Ruslan I; Rosales, Ivy A; Fisher, David E; Gupta, Navin; Morizane, Ryuji; Greka, Anna; Pike, J Wesley; Mannstadt, Michael; Wein, Marc N | The Journal of clinical investigation | 36862513 | 2023 Mar 02 |
Nanoparticle stereochemistry-dependent endocytic processing improves in vivo mRNA delivery. | Hatit, Marine Z C; Dobrowolski, Curtis N; Lokugamage, Melissa P; Loughrey, David; Ni, Huanzhen; Zurla, Chiara; Da Silva Sanchez, Alejandro J; Radmand, Afsane; Huayamares, Sebastian G; Zenhausern, Ryan; Paunovska, Kalina; Peck, Hannah E; Kim, Jinwhan; Sato, Manaka; Feldman, Jacob I; Rivera, Michael-Alexander; Cristian, Ana; Kim, YongTae; Santangelo, Philip J; Dahlman, James E | Nature chemistry | 36864143 | 2023 Apr |
The history, use, and challenges of therapeutic somatic cell and germline gene editing. | Ryu, Junghyun; Adashi, Eli Y; Hennebold, Jon D | Fertility and sterility | 36878350 | 2023 Sep |
ATP/ADP biosensor organoids for drug nephrotoxicity assessment. | Susa, Koichiro; Kobayashi, Kenichi; Galichon, Pierre; Matsumoto, Takuya; Tamura, Akitoshi; Hiratsuka, Ken; Gupta, Navin R; Yazdi, Iman K; Bonventre, Joseph V; Morizane, Ryuji | Frontiers in cell and developmental biology | 36936695 | 2023 |
Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing. | McAuley, Grace E; Yiu, Gloria; Chang, Patrick C; Newby, Gregory A; Campo-Fernandez, Beatriz; Fitz-Gibbon, Sorel T; Wu, Xiaomeng; Kang, Sung-Hae L; Garibay, Amber; Butler, Jeffrey; Christian, Valentina; Wong, Ryan L; Everette, Kelcee A; Azzun, Anthony; Gelfer, Hila; Seet, Christopher S; Narendran, Aru; Murguia-Favela, Luis; Romero, Zulema; Wright, Nicola; Liu, David R; Crooks, Gay M; Kohn, Donald B | Cell | 36944331 | 2023 Mar 30 |
Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities. | Kar, Bibekananda; Castillo, Santiago R; Sabharwal, Ankit; Clark, Karl J; Ekker, Stephen C | International journal of molecular sciences | 36982871 | 2023 Mar 18 |
Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo. | Zhang, Han; Kelly, Karen; Lee, Jonathan; Echeverria, Dimas; Cooper, David; Panwala, Rebecca; Chen, Zexiang; Gaston, Nicholas; Newby, Gregory A; Xie, Jun; Liu, David R; Gao, Guangping; Wolfe, Scot A; Khvorova, Anastasia; Watts, Jonathan K; Sontheimer, Erik J | bioRxiv : the preprint server for biology | 36993169 | 2023 Mar 23 |
Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells. | Tsuchida, Connor A; Brandes, Nadav; Bueno, Raymund; Trinidad, Marena; Mazumder, Thomas; Yu, Bingfei; Hwang, Byungjin; Chang, Christopher; Liu, Jamin; Sun, Yang; Hopkins, Caitlin R; Parker, Kevin R; Qi, Yanyan; Satpathy, Ansuman T; Stadtmauer, Edward A; Cate, Jamie H D; Eyquem, Justin; Fraietta, Joseph A; June, Carl H; Chang, Howard Y; Ye, Chun Jimmie; Doudna, Jennifer A | bioRxiv : the preprint server for biology | 36993359 | 2023 Mar 22 |
Base editing rescue of spinal muscular atrophy in cells and in mice. | Arbab, Mandana; Matuszek, Zaneta; Kray, Kaitlyn M; Du, Ailing; Newby, Gregory A; Blatnik, Anton J; Raguram, Aditya; Richter, Michelle F; Zhao, Kevin T; Levy, Jonathan M; Shen, Max W; Arnold, W David; Wang, Dan; Xie, Jun; Gao, Guangping; Burghes, Arthur H M; Liu, David R | Science (New York, N.Y.) | 36996170 | 2023 Apr 21 |
Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing. | Li, Bowen; Manan, Rajith Singh; Liang, Shun-Qing; Gordon, Akiva; Jiang, Allen; Varley, Andrew; Gao, Guangping; Langer, Robert; Xue, Wen; Anderson, Daniel | Nature biotechnology | 36997680 | 2023 Oct |
Rescue of GM3 synthase deficiency by spatially controlled, rAAV-mediated ST3GAL5 delivery. | Yang, Huiya; Brown Jr, Robert H; Wang, Dan; Strauss, Kevin A; Gao, Guangping | JCI insight | 37014712 | 2023 May 08 |
Adeno-Associated virus 8 delivers an immunomodulatory peptide to mouse liver more efficiently than to rat liver. | Wang, Yuqing; Hurley, Ayrea; De Giorgi, Marco; Tanner, Mark R; Hu, Rong-Chi; Pennington, Michael W; Lagor, William R; Beeton, Christine | PloS one | 37040361 | 2023 |
CaMKIIδ gene editing- A base hit for the heart. | Walkey, Christopher J; Lagor, William R | The journal of cardiovascular aging | 37065676 | 2023 |
Reprogramming by drug-like molecules leads to regeneration of cochlear hair cell-like cells in adult mice. | Quan, Yi-Zhou; Wei, Wei; Ergin, Volkan; Rameshbabu, Arun Prabhu; Huang, Mingqian; Tian, Chunjie; Saladi, Srinivas Vinod; Indzhykulian, Artur A; Chen, Zheng-Yi | Proceedings of the National Academy of Sciences of the United States of America | 37068229 | 2023 Apr 25 |
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice. | Everette, Kelcee A; Newby, Gregory A; Levine, Rachel M; Mayberry, Kalin; Jang, Yoonjeong; Mayuranathan, Thiyagaraj; Nimmagadda, Nikitha; Dempsey, Erin; Li, Yichao; Bhoopalan, Senthil Velan; Liu, Xiong; Davis, Jessie R; Nelson, Andrew T; Chen, Peter J; Sousa, Alexander A; Cheng, Yong; Tisdale, John F; Weiss, Mitchell J; Yen, Jonathan S; Liu, David R | Nature biomedical engineering | 37069266 | 2023 May |
Strategies for Improving Vascularization in Kidney Organoids: A Review of Current Trends. | Konoe, Ran; Morizane, Ryuji | Biology | 37106704 | 2023 Mar 26 |
HIV-Differentiated Metabolite N-Acetyl-L-Alanine Dysregulates Human Natural Killer Cell Responses to Mycobacterium tuberculosis Infection. | Yang, Baojun; Mukherjee, Tanmoy; Radhakrishnan, Rajesh; Paidipally, Padmaja; Ansari, Danish; John, Sahana; Vankayalapati, Ramakrishna; Tripathi, Deepak; Yi, Guohua | International journal of molecular sciences | 37108430 | 2023 Apr 14 |
Massively parallel base editing to map variant effects in human hematopoiesis. | Martin-Rufino, Jorge D; Castano, Nicole; Pang, Michael; Grody, Emanuelle I; Joubran, Samantha; Caulier, Alexis; Wahlster, Lara; Li, Tongqing; Qiu, Xiaojie; Riera-Escandell, Anna Maria; Newby, Gregory A; Al'Khafaji, Aziz; Chaudhary, Santosh; Black, Susan; Weng, Chen; Munson, Glen; Liu, David R; Wlodarski, Marcin W; Sims, Kacie; Oakley, Jamie H; Fasano, Ross M; Xavier, Ramnik J; Lander, Eric S; Klein, Daryl E; Sankaran, Vijay G | Cell | 37137305 | 2023 May 25 |
Efficient prime editing in mouse brain, liver and heart with dual AAVs. | Davis, Jessie R; Banskota, Samagya; Levy, Jonathan M; Newby, Gregory A; Wang, Xiao; Anzalone, Andrew V; Nelson, Andrew T; Chen, Peter J; Hennes, Andrew D; An, Meirui; Roh, Heejin; Randolph, Peyton B; Musunuru, Kiran; Liu, David R | Nature biotechnology | 37142705 | 2023 May 04 |
Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes. | Foss, Dana V; Muldoon, Joseph J; Nguyen, David N; Carr, Daniel; Sahu, Srishti U; Hunsinger, John M; Wyman, Stacia K; Krishnappa, Netravathi; Mendonsa, Rima; Schanzer, Elaine V; Shy, Brian R; Vykunta, Vivasvan S; Allain, Vincent; Li, Zhongmei; Marson, Alexander; Eyquem, Justin; Wilson, Ross C | Nature biomedical engineering | 37147433 | 2023 May |
Renewal of oligodendrocyte lineage reverses dysmyelination and CNS neurodegeneration through corrected N-acetylaspartate metabolism. | Lotun, Anoushka; Li, Danning; Xu, Hongxia; Su, Qin; Tuncer, Serafettin; Sanmiguel, Julio; Mooney, Morgan; Baer, Christina E; Ulbrich, Russell; Eyles, Stephen J; Strittmatter, Lara; Hayward, Lawrence J; Gessler, Dominic J; Gao, Guangping | Progress in neurobiology | 37149081 | 2023 Jul |
Genome-wide profiling of prime editor off-target sites in vitro and in vivo using PE-tag. | Liang, Shun-Qing; Liu, Pengpeng; Ponnienselvan, Karthikeyan; Suresh, Sneha; Chen, Zexiang; Kramme, Christian; Chatterjee, Pranam; Zhu, Lihua Julie; Sontheimer, Erik J; Xue, Wen; Wolfe, Scot A | Nature methods | 37156841 | 2023 Jun |
A Multifunctional and Highly Adaptable Reporter System for CRISPR/Cas Editing. | Wettengel, Jochen M; Hansen-Palmus, Lea; Yusova, Sofiya; Rust, Lauren; Biswas, Sreya; Carson, Julien; Ryu, Junghyun; Bimber, Benjamin N; Hennebold, Jon D; Burwitz, Benjamin J | International journal of molecular sciences | 37175977 | 2023 May 05 |
Genetic background in the rat affects endocrine and metabolic outcomes of bisphenol F exposure. | Wagner, Valerie A; Holl, Katie L; Clark, Karen C; Reho, John J; Dwinell, Melinda R; Lehmler, Hans-Joachim; Raff, Hershel; Grobe, Justin L; Kwitek, Anne E | Toxicological sciences : an official journal of the Society of Toxicology | 37191987 | 2023 Jun 28 |
Orthogonal CRISPR screens to identify transcriptional and epigenetic regulators of human CD8 T cell function. | McCutcheon, Sean R; Swartz, Adam M; Brown, Michael C; Barrera, Alejandro; Amador, Christian McRoberts; Siklenka, Keith; Humayun, Lucas; Isaacs, James M; Reddy, Timothy E; Nair, Smita; Antonia, Scott; Gersbach, Charles A | bioRxiv : the preprint server for biology | 37205457 | 2023 May 01 |
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8. | Du, Wan; Ergin, Volkan; Loeb, Corena; Huang, Mingqian; Silver, Stewart; Armstrong, Ariel Miura; Huang, Zaohua; Gurumurthy, Channabasavaiah B; Staecker, Hinrich; Liu, Xuezhong; Chen, Zheng-Yi | Molecular therapy : the journal of the American Society of Gene Therapy | 37244253 | 2023 May 22 |
Reducing the inherent auto-inhibitory interaction within the pegRNA enhances prime editing efficiency. | Ponnienselvan, Karthikeyan; Liu, Pengpeng; Nyalile, Thomas; Oikemus, Sarah; Maitland, Stacy A; Lawson, Nathan D; Luban, Jeremy; Wolfe, Scot A | Nucleic acids research | 37246708 | 2023 Jul 21 |
Liver directed adeno-associated viral vectors to treat metabolic disease. | Chuecos, Marcel A; Lagor, William R | Journal of inherited metabolic disease | 37254440 | 2023 May 30 |
Template-jumping prime editing enables large insertion and exon rewriting in vivo. | Zheng, Chunwei; Liu, Bin; Dong, Xiaolong; Gaston, Nicholas; Sontheimer, Erik J; Xue, Wen | Nature communications | 37291100 | 2023 Jun 08 |
Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing. | Brooks, Dominique L; Carrasco, Manuel J; Qu, Ping; Peranteau, William H; Ahrens-Nicklas, Rebecca C; Musunuru, Kiran; Alameh, Mohamad-Gabriel; Wang, Xiao | Nature communications | 37301931 | 2023 Jun 10 |
Mercapto-pyrimidines are reversible covalent inhibitors of the papain-like protease (PLpro) and inhibit SARS-CoV-2 (SCoV-2) replication. | Bajaj, Teena; Wehri, Eddie; Suryawanshi, Rahul K; King, Elizabeth; Pardeshi, Kundan Singh; Behrouzi, Kamyar; Khodabakhshi, Zahra; Schulze-Gahmen, Ursula; Kumar, G Renuka; Mofrad, Mohammad R K; Nomura, Daniel K; Ott, Melanie; Schaletzky, Julia; Murthy, Niren | RSC advances | 37312993 | 2023 Jun 09 |
Scalable projected Light Sheet Microscopy for high-resolution imaging of large samples. | Chen, Yannan; Gong, Cheng; Chauhan, Shradha; De La Cruz, Estanislao Daniel; Datta, Malika S; Tomer, Raju | bioRxiv : the preprint server for biology | 37333196 | 2023 Jun 05 |
Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing. | Yip, Mitchell; Chen, Jing; Zhi, Yan; Tran, Ngoc Tam; Namkung, Suk; Pastor, Eric; Gao, Guangping; Tai, Phillip W L | Viruses | 37376529 | 2023 May 24 |
Utilizing directed evolution to interrogate and optimize CRISPR/Cas guide RNA scaffolds. | Bush, Korie; Corsi, Giulia I; Yan, Amy C; Haynes, Keith; Layzer, Juliana M; Zhou, Jonathan H; Llanga, Telmo; Gorodkin, Jan; Sullenger, Bruce A | Cell chemical biology | 37390831 | 2023 Aug 17 |
Potent and uniform fetal hemoglobin induction via base editing. | Mayuranathan, Thiyagaraj; Newby, Gregory A; Feng, Ruopeng; Yao, Yu; Mayberry, Kalin D; Lazzarotto, Cicera R; Li, Yichao; Levine, Rachel M; Nimmagadda, Nikitha; Dempsey, Erin; Kang, Guolian; Porter, Shaina N; Doerfler, Phillip A; Zhang, Jingjing; Jang, Yoonjeong; Chen, Jingjing; Bell, Henry W; Crossley, Merlin; Bhoopalan, Senthil Velan; Sharma, Akshay; Tisdale, John F; Pruett-Miller, Shondra M; Cheng, Yong; Tsai, Shengdar Q; Liu, David R; Weiss, Mitchell J; Yen, Jonathan S | Nature genetics | 37400614 | 2023 Jul |
Rational nanoparticle design: Optimization using insights from experiments and mathematical models. | Richfield, Owen; Piotrowski-Daspit, Alexandra S; Shin, Kwangsoo; Saltzman, W Mark | Journal of controlled release : official journal of the Controlled Release Society | 37442201 | 2023 Aug |
Adenoviral vectors infect B lymphocytes in vivo. | Rice-Boucher, Paul J; Mendonça, Samir Andrade; Alvarez, Aluet Borrego; Sturtz, Alexandria J; Lorincz, Reka; Dmitriev, Igor P; Kashentseva, Elena A; Lu, Zhi Hong; Romano, Rosa; Selby, Mark; Pingale, Kunal; Curiel, David T | Molecular therapy : the journal of the American Society of Gene Therapy | 37452494 | 2023 Sep 06 |
Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier. | Huang, Qin; Chen, Albert T; Chan, Ken Y; Sorensen, Hikari; Barry, Andrew J; Azari, Bahar; Zheng, Qingxia; Beddow, Thomas; Zhao, Binhui; Tobey, Isabelle G; Moncada-Reid, Cynthia; Eid, Fatma-Elzahraa; Walkey, Christopher J; Ljungberg, M Cecilia; Lagor, William R; Heaney, Jason D; Chan, Yujia A; Deverman, Benjamin E | PLoS biology | 37467291 | 2023 Jul |
Gene editing therapeutics based on mRNA delivery. | Popovitz, Juliana; Sharma, Rohit; Hoshyar, Reyhane; Soo Kim, Beob; Murthy, Niren; Lee, Kunwoo | Advanced drug delivery reviews | 37516409 | 2023 Sep |
Individually tailored spatial-spectral pulsed CEST MRI for ratiometric mapping of myocardial energetic species at 3T. | Ayala, Cindy; Luo, Huiwen; Godines, Kevin; Alghuraibawi, Wissam; Ahn, Sinyeob; Rehwald, Wolfgang; Grissom, William A; Vandsburger, Moriel H | Magnetic resonance in medicine | 37526176 | 2023 Dec |
In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene. | De Giorgi, Marco; Park, So Hyun; Castoreno, Adam; Cao, Mingming; Hurley, Ayrea; Saxena, Lavanya; Chuecos, Marcel A; Walkey, Christopher J; Doerfler, Alexandria M; Furgurson, Mia N; Ljungberg, M Cecilia; Patel, Kalyani R; Hyde, Sarah; Chickering, Tyler; Lefebvre, Stephanie; Wassarman, Kelly; Miller, Patrick; Qin, June; Schlegel, Mark K; Zlatev, Ivan; Li, Rich Gang; Kim, Jong; Martin, James F; Bissig, Karl-Dimiter; Jadhav, Vasant; Bao, Gang; Lagor, William R | bioRxiv : the preprint server for biology | 37546995 | 2023 Jul 29 |
Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells. | Joynt, Anya T; Kavanagh, Erin W; Newby, Gregory A; Mitchell, Shakela; Eastman, Alice C; Paul, Kathleen C; Bowling, Alyssa D; Osorio, Derek L; Merlo, Christian A; Patel, Shivani U; Raraigh, Karen S; Liu, David R; Sharma, Neeraj; Cutting, Garry R | Molecular therapy. Nucleic acids | 37547293 | 2023 Sep 12 |
Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy. | Kabra, Meha; Shahi, Pawan K; Wang, Yuyuan; Sinha, Divya; Spillane, Allison; Newby, Gregory A; Saxena, Shivani; Tong, Yao; Chang, Yu; Abdeen, Amr A; Edwards, Kimberly L; Theisen, Cole O; Liu, David R; Gamm, David M; Gong, Shaoqin; Saha, Krishanu; Pattnaik, Bikash R | The Journal of clinical investigation | 37561581 | 2023 Oct 02 |
Focused ultrasound-mediated brain genome editing. | Lao, Yeh-Hsing; Ji, Robin; Zhou, Joyce K; Snow, Kathy J; Kwon, Nancy; Saville, Ethan; He, Siyu; Chauhan, Shradha; Chi, Chun-Wei; Datta, Malika S; Zhang, Hairong; Quek, Chai Hoon; Cai, S Sarah; Li, Mingqiang; Gaitan, Yaned; Bechtel, Lawrence; Wu, Shih-Ying; Lutz, Cathleen M; Tomer, Raju; Murray, Stephen A; Chavez, Alejandro; Konofagou, Elisa E; Leong, Kam W | Proceedings of the National Academy of Sciences of the United States of America | 37579143 | 2023 Aug 22 |
CFTR-rich ionocytes mediate chloride absorption across airway epithelia. | Lei, Lei; Traore, Soumba; Romano Ibarra, Guillermo S; Karp, Philip H; Rehman, Tayyab; Meyerholz, David K; Zabner, Joseph; Stoltz, David A; Sinn, Patrick L; Welsh, Michael J; McCray Jr, Paul B; Thornell, Ian M | The Journal of clinical investigation | 37581935 | 2023 Oct 16 |
Nonviral In Vivo Delivery of CRISPR-Cas9 Using Protein-Agnostic, High-Loading Porous Silicon and Polymer Nanoparticles. | Fletcher, R Brock; Stokes, Larry D; Kelly 3rd, Isom B; Henderson, Katelyn M; Vallecillo-Viejo, Isabel C; Colazo, Juan M; Wong, Benjamin V; Yu, Fang; d'Arcy, Richard; Struthers, Morgan N; Evans, Brian C; Ayers, Jacob; Castanon, Matthew; Weirich, Michael J; Reilly, Sarah K; Patel, Shrusti S; Ivanova, Yoanna I; Silvera Batista, Carlos A; Weiss, Sharon M; Gersbach, Charles A; Brunger, Jonathan M; Duvall, Craig L | ACS nano | 37582231 | 2023 Sep 12 |
Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo. | Tao, Yong; Lamas, Veronica; Du, Wan; Zhu, Wenliang; Li, Yiran; Whittaker, Madelynn N; Zuris, John A; Thompson, David B; Rameshbabu, Arun Prabhu; Shu, Yilai; Gao, Xue; Hu, Johnny H; Pei, Charles; Kong, Wei-Jia; Liu, Xuezhong; Wu, Hao; Kleinstiver, Benjamin P; Liu, David R; Chen, Zheng-Yi | Nature communications | 37582836 | 2023 Aug 15 |
Polymer nanoparticles deliver mRNA to the lung for mucosal vaccination. | Suberi, Alexandra; Grun, Molly K; Mao, Tianyang; Israelow, Benjamin; Reschke, Melanie; Grundler, Julian; Akhtar, Laiba; Lee, Teresa; Shin, Kwangsoo; Piotrowski-Daspit, Alexandra S; Homer, Robert J; Iwasaki, Akiko; Suh, Hee-Won; Saltzman, W Mark | Science translational medicine | 37585505 | 2023 Aug 16 |
Phage-assisted evolution and protein engineering yield compact, efficient prime editors. | Doman, Jordan L; Pandey, Smriti; Neugebauer, Monica E; An, Meirui; Davis, Jessie R; Randolph, Peyton B; McElroy, Amber; Gao, Xin D; Raguram, Aditya; Richter, Michelle F; Everette, Kelcee A; Banskota, Samagya; Tian, Kathryn; Tao, Y Allen; Tolar, Jakub; Osborn, Mark J; Liu, David R | Cell | 37657419 | 2023 Aug 31 |
Trichostatin A for Efficient CRISPR-Cas9 Gene Editing of Human Pluripotent Stem Cells. | Molugu, Kaivalya; Khajanchi, Namita; Lazzarotto, Cicera R; Tsai, Shengdar Q; Saha, Krishanu | The CRISPR journal | 37676985 | 2023 Oct |
Lipid Nanoparticles Deliver mRNA to the Brain after an Intracerebral Injection. | Tuma, Jan; Chen, Yu-Ju; Collins, Michael G; Paul, Abhik; Li, Jie; Han, Hesong; Sharma, Rohit; Murthy, Niren; Lee, Hye Young | Biochemistry | 37729550 | 2023 Dec 19 |
Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery. | Gonzalez, Trevor J; Mitchell-Dick, Aaron; Blondel, Leo O; Fanous, Marco M; Hull, Joshua A; Oh, Daniel K; Moller-Tank, Sven; Castellanos Rivera, Ruth M; Piedrahita, Jorge A; Asokan, Aravind | Nature protocols | 37735235 | 2023 Nov |
A brown fat-enriched adipokine, ASRA, is a leptin receptor antagonist that stimulates appetite. | Huang, Lei; Liu, Pengpeng; Du, Yong; Pan, Dongning; Lee, Alexandra; Wolfe, Scot A; Wang, Yong-Xu | bioRxiv : the preprint server for biology | 37745491 | 2023 Sep 12 |
Navigating the kidney organoid: insights into assessment and enhancement of nephron function. | Tabibzadeh, Nahid; Satlin, Lisa M; Jain, Sanjay; Morizane, Ryuji | American journal of physiology. Renal physiology | 37767571 | 2023 Dec 01 |
Assigning functionality to cysteines by base editing of cancer dependency genes. | Li, Haoxin; Ma, Tiantai; Remsberg, Jarrett R; Won, Sang Joon; DeMeester, Kristen E; Njomen, Evert; Ogasawara, Daisuke; Zhao, Kevin T; Huang, Tony P; Lu, Bingwen; Simon, Gabriel M; Melillo, Bruno; Schreiber, Stuart L; Lykke-Andersen, Jens; Liu, David R; Cravatt, Benjamin F | Nature chemical biology | 37783940 | 2023 Nov |
Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells. | Tsuchida, Connor A; Brandes, Nadav; Bueno, Raymund; Trinidad, Marena; Mazumder, Thomas; Yu, Bingfei; Hwang, Byungjin; Chang, Christopher; Liu, Jamin; Sun, Yang; Hopkins, Caitlin R; Parker, Kevin R; Qi, Yanyan; Hofman, Laura; Satpathy, Ansuman T; Stadtmauer, Edward A; Cate, Jamie H D; Eyquem, Justin; Fraietta, Joseph A; June, Carl H; Chang, Howard Y; Ye, Chun Jimmie; Doudna, Jennifer A | Cell | 37794590 | 2023 Oct 12 |
Redirecting AAV vectors to extrahepatic tissues. | Asokan, Aravind; Shen, Shen | Molecular therapy : the journal of the American Society of Gene Therapy | 37805712 | 2023 Dec 06 |
A marmoset brain cell census reveals regional specialization of cellular identities. | Krienen, Fenna M; Levandowski, Kirsten M; Zaniewski, Heather; Del Rosario, Ricardo C H; Schroeder, Margaret E; Goldman, Melissa; Wienisch, Martin; Lutservitz, Alyssa; Beja-Glasser, Victoria F; Chen, Cindy; Zhang, Qiangge; Chan, Ken Y; Li, Katelyn X; Sharma, Jitendra; McCormack, Dana; Shin, Tay Won; Harrahill, Andrew; Nyase, Eric; Mudhar, Gagandeep; Mauermann, Abigail; Wysoker, Alec; Nemesh, James; Kashin, Seva; Vergara, Josselyn; Chelini, Gabriele; Dimidschstein, Jordane; Berretta, Sabina; Deverman, Benjamin E; Boyden, Ed; McCarroll, Steven A; Feng, Guoping | Science advances | 37824615 | 2023 Oct 13 |
Avian influenza A viruses exhibit plasticity in sialylglycoconjugate receptor usage in human lung cells. | Liang, Chieh-Yu; Huang, Iris; Han, Julianna; Sownthirarajan, Boopathi; Kulhankova, Katarina; Murray, Nathan B; Taherzadeh, Mehrnoush; Archer-Hartmann, Stephanie; Pepi, Lauren; Manivasagam, Senthamizharasi; Plung, Jesse; Sturtz, Miranda; Yu, Yolanda; Vogel, Olivia A; Kandasamy, Matheswaran; Gourronc, Francoise A; Klingelhutz, Aloysius J; Choudhury, Biswa; Rong, Lijun; Perez, Jasmine T; Azadi, Parastoo; McCray Jr, Paul B; Neelamegham, Sriram; Manicassamy, Balaji | Journal of virology | 37843369 | 2023 Nov 30 |
PCSK9 activation promotes early atherosclerosis in a vascular microphysiological system. | Lee, Jounghyun H; Shores, Kevin L; Breithaupt, Jason J; Lee, Caleb S; Fodera, Daniella M; Kwon, Jennifer B; Ettyreddy, Adarsh R; Myers, Kristin M; Evison, Benny J; Suchowerska, Alexandra K; Gersbach, Charles A; Leong, Kam W; Truskey, George A | APL bioengineering | 37854060 | 2023 Dec |
The Power of the Heterogeneous Stock Rat Founder Strains in Modeling Metabolic Disease. | Wagner, Valerie A; Holl, Katie L; Clark, Karen C; Reho, John J; Lehmler, Hans-Joachim; Wang, Kai; Grobe, Justin L; Dwinell, Melinda R; Raff, Hershel; Kwitek, Anne E | Endocrinology | 37882530 | 2023 Nov 02 |
Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice. | Chang, Hao; Du, Ailing; Jiang, Jun; Ren, Lingzhi; Liu, Nan; Zhou, Xuntao; Liang, Jialing; Gao, Guangping; Wang, Dan | Molecular therapy. Methods & clinical development | 37886602 | 2023 Dec 14 |
CasPEDIA Database: a functional classification system for class 2 CRISPR-Cas enzymes. | Adler, Benjamin A; Trinidad, Marena I; Bellieny-Rabelo, Daniel; Zhang, Elaine; Karp, Hannah M; Skopintsev, Petr; Thornton, Brittney W; Weissman, Rachel F; Yoon, Peter H; Chen, LinXing; Hessler, Tomas; Eggers, Amy R; Colognori, David; Boger, Ron; Doherty, Erin E; Tsuchida, Connor A; Tran, Ryan V; Hofman, Laura; Shi, Honglue; Wasko, Kevin M; Zhou, Zehan; Xia, Chenglong; Al-Shimary, Muntathar J; Patel, Jaymin R; Thomas, Vienna C J X; Pattali, Rithu; Kan, Matthew J; Vardapetyan, Anna; Yang, Alana; Lahiri, Arushi; Maxwell, Micaela F; Murdock, Andrew G; Ramit, Glenn C; Henderson, Hope R; Calvert, Roland W; Bamert, Rebecca S; Knott, Gavin J; Lapinaite, Audrone; Pausch, Patrick; Cofsky, Joshua C; Sontheimer, Erik J; Wiedenheft, Blake; Fineran, Peter C; Brouns, Stan J J; Sashital, Dipali G; Thomas, Brian C; Brown, Christopher T; Goltsman, Daniela S A; Barrangou, Rodolphe; Siksnys, Virginius; Banfield, Jillian F; Savage, David F; Doudna, Jennifer A | Nucleic acids research | 37889041 | 2024 Jan 05 |
DNA recognition and induced genome modification by a hydroxymethyl-γ tail-clamp peptide nucleic acid. | Oyaghire, Stanley N; Quijano, Elias; Perera, J Dinithi R; Mandl, Hanna K; Saltzman, W Mark; Bahal, Raman; Glazer, Peter M | Cell reports. Physical science | 37920723 | 2023 Oct 18 |
Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need. | Brooks, Dominique L; Whittaker, Madelynn N; Qu, Ping; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C; Wang, Xiao | American journal of human genetics | 37924808 | 2023 Dec 07 |
Building blocks for better biorepositories in Africa. | Croxton, Talishiea; Jonathan, Emmanuel; Suleiman, Kareemah; Balogun, Olasinbo; Ozumba, Petronilla J; Aloyo, Sharley M; Nsubuga, Gideon; Kamulegeya, Rogers E; Newton, Lwanga; Mukisa, John; Kader, Mukthar; Damaneite, Vuyo; Nadoma, Sunji; Onyemata, Ezenwa James; Anzaku, Abbas Abel; Nasinghe, Emmanuel; Troyer, Jennifer; Joubert, Bonnie R; Beiswanger, Christine; Joloba, Moses L; Mayne, Elizabeth; Abimiku, Alash'le; H3Africa Biorepository PI Working Group | Genome medicine | 37932809 | 2023 Nov 06 |
Human red blood cells release microvesicles with distinct sizes and protein composition that alter neutrophil phagocytosis. | de Oliveira Junior, Getulio Pereira; Welsh, Joshua A; Pinckney, Brandy; Palu, Cintia C; Lu, Shulin; Zimmerman, Alan; Barbosa, Raquel Hora; Sahu, Parul; Noshin, Maeesha; Gummuluru, Suryaram; Tigges, John; Jones, Jennifer Clare; Ivanov, Alexander R; Ghiran, Ionita C | Journal of extracellular biology | 37942280 | 2023 Nov |
Transcriptional and epigenetic regulators of human CD8+ T cell function identified through orthogonal CRISPR screens. | McCutcheon, Sean R; Swartz, Adam M; Brown, Michael C; Barrera, Alejandro; McRoberts Amador, Christian; Siklenka, Keith; Humayun, Lucas; Ter Weele, Maria A; Isaacs, James M; Reddy, Timothy E; Allen, Andrew S; Nair, Smita K; Antonia, Scott J; Gersbach, Charles A | Nature genetics | 37945901 | 2023 Dec |
Eukaryotic RNA-guided endonucleases evolved from a unique clade of bacterial enzymes. | Yoon, Peter H; Skopintsev, Petr; Shi, Honglue; Chen, LinXing; Adler, Benjamin A; Al-Shimary, Muntathar; Craig, Rory J; Loi, Kenneth J; DeTurk, Evan C; Li, Zheng; Amerasekera, Jasmine; Trinidad, Marena; Nisonoff, Hunter; Chen, Kai; Lahiri, Arushi; Boger, Ron; Jacobsen, Steve; Banfield, Jillian F; Doudna, Jennifer A | Nucleic acids research | 37971304 | 2023 Dec 11 |
Combinatorial development of nebulized mRNA delivery formulations for the lungs. | Jiang, Allen Y; Witten, Jacob; Raji, Idris O; Eweje, Feyisayo; MacIsaac, Corina; Meng, Sabrina; Oladimeji, Favour A; Hu, Yizong; Manan, Rajith S; Langer, Robert; Anderson, Daniel G | Nature nanotechnology | 37985700 | 2023 Nov 20 |
Genomic characterization of SARS-CoV-2 from Uganda using MinION nanopore sequencing. | Kia, Praiscillia; Katagirya, Eric; Kakembo, Fredrick Elishama; Adera, Doreen Ato; Nsubuga, Moses Luutu; Yiga, Fahim; Aloyo, Sharley Melissa; Aujat, Brendah Ronah; Anguyo, Denis Foe; Katabazi, Fred Ashaba; Kigozi, Edgar; Joloba, Moses L; Kateete, David Patrick | Scientific reports | 37993530 | 2023 Nov 22 |
Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo. | Zhang, Han; Kelly, Karen; Lee, Jonathan; Echeverria, Dimas; Cooper, David; Panwala, Rebecca; Amrani, Nadia; Chen, Zexiang; Gaston, Nicholas; Wagh, Atish; Newby, Gregory A; Xie, Jun; Liu, David R; Gao, Guangping; Wolfe, Scot A; Khvorova, Anastasia; Watts, Jonathan K; Sontheimer, Erik J | Nucleic acids research | 38033325 | 2023 Nov 30 |
Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo. | Kulhankova, Katarina; Traore, Soumba; Cheng, Xue; Benk-Fortin, Hadrien; Hallée, Stéphanie; Harvey, Mario; Roberge, Joannie; Couture, Frédéric; Kohli, Sajeev; Gross, Thomas J; Meyerholz, David K; Rettig, Garrett R; Thommandru, Bernice; Kurgan, Gavin; Wohlford-Lenane, Christine; Hartigan-O'Connor, Dennis J; Yates, Bradley P; Newby, Gregory A; Liu, David R; Tarantal, Alice F; Guay, David; McCray Jr, Paul B | Nature communications | 38052872 | 2023 Dec 05 |
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy. | Alves, Christiano R R; Ha, Leillani L; Yaworski, Rebecca; Sutton, Emma R; Lazzarotto, Cicera R; Christie, Kathleen A; Reilly, Aoife; Beauvais, Ariane; Doll, Roman M; de la Cruz, Demitri; Maguire, Casey A; Swoboda, Kathryn J; Tsai, Shengdar Q; Kothary, Ras | Nature biomedical engineering | 38057426 | 2024 Feb |
Development of a human glioblastoma model using humanized DRAG mice for immunotherapy. | Srivastava, Rashmi; Labani-Motlagh, Alireza; Chen, Apeng; Bohorquez, Jose Alejandro; Qin, Bin; Dodda, Meghana; Yang, Fan; Ansari, Danish; Patel, Sahil; Ji, Honglong; Trasti, Scott; Chao, Yapeng; Patel, Yash; Zou, Han; Hu, Baoli; Yi, Guohua | Antibody therapeutics | 38075240 | 2023 Oct |
Harnessing non-Watson-Crick's base pairing to enhance CRISPR effectors cleavage activities and enable gene editing in mammalian cells. | Gao, Shuliang; Guan, Huiwen; Bloomer, Hanan; Wich, Douglas; Song, Donghui; Khirallah, Jennifer; Ye, Zhongfeng; Zhao, Yu; Chen, Mengting; Xu, Chutian; Liu, Lihan; Xu, Qiaobing | Proceedings of the National Academy of Sciences of the United States of America | 38150477 | 2024 Jan 09 |
Tumor-tropic Trojan horses: Using mesenchymal stem cells as cellular nanotheranostics. | Rosu, Ana; Ghaemi, Behnaz; Bulte, Jeff W M; Shakeri-Zadeh, Ali | Theranostics | 38169524 | 2024 |
Protein-based nanoparticles for therapeutic nucleic acid delivery. | Eweje, Feyisayo; Walsh, Michelle L; Ahmad, Kiran; Ibrahim, Vanessa; Alrefai, Assma; Chen, Jiaxuan; Chaikof, Elliot L | Biomaterials | 38181574 | 2024 Mar |
An AAV capsid reprogrammed to bind human Transferrin Receptor mediates brain-wide gene delivery. | Huang, Qin; Chan, Ken Y; Lou, Shan; Keyes, Casey; Wu, Jason; Botticello-Romero, Nuria R; Zheng, Qingxia; Johnston, Jencilin; Mills, Allan; Brauer, Pamela P; Clouse, Gabrielle; Pacouret, Simon; Harvey, John W; Beddow, Thomas; Hurley, Jenna K; Tobey, Isabelle G; Powell, Megan; Chen, Albert T; Barry, Andrew J; Eid, Fatma-Elzahraa; Chan, Yujia A; Deverman, Benjamin E | bioRxiv : the preprint server for biology | 38187643 | 2023 Dec 22 |
Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo. | An, Meirui; Raguram, Aditya; Du, Samuel W; Banskota, Samagya; Davis, Jessie R; Newby, Gregory A; Chen, Paul Z; Palczewski, Krzysztof; Liu, David R | Nature biotechnology | 38191664 | 2024 Jan 08 |
Validated assays for the quantification of C9orf72 human pathology. | Salomonsson, S E; Maltos, A M; Gill, K; Aladesuyi Arogundade, O; Brown, K A; Sachdev, A; Sckaff, M; Lam, K J K; Fisher, I J; Chouhan, R S; Van Laar, V S; Marley, C B; McLaughlin, I; Bankiewicz, K S; Tsai, Y-C; Conklin, B R; Clelland, C D | Scientific reports | 38191789 | 2024 Jan 08 |
Mechanosensitive genomic enhancers potentiate the cellular response to matrix stiffness. | Cosgrove, Brian D; Bounds, Lexi R; Taylor, Carson Key; Su, Alan L; Rizzo, Anthony J; Barrera, Alejandro; Crawford, Gregory E; Hoffman, Brenton D; Gersbach, Charles A | bioRxiv : the preprint server for biology | 38260455 | 2024 Jan 10 |
AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfecta. | Yang, Yeon-Suk; Sato, Tadatoshi; Chaugule, Sachin; Ma, Hong; Xie, Jun; Gao, Guangping; Shim, Jae-Hyuck | Molecular therapy. Nucleic acids | 38261950 | 2024 Mar 12 |
Advancements in therapeutic development: kidney organoids and organs on a chip. | Tabibzadeh, Nahid; Morizane, Ryuji | Kidney international | 38296026 | 2024 Apr |
Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference. | Zhang, Emily; Neugebauer, Monica E; Krasnow, Nicholas A; Liu, David R | Nature communications | 38402281 | 2024 Feb 24 |
Cationic cholesterol-dependent LNP delivery to lung stem cells, the liver, and heart. | Radmand, Afsane; Kim, Hyejin; Beyersdorf, Jared; Dobrowolski, Curtis N; Zenhausern, Ryan; Paunovska, Kalina; Huayamares, Sebastian G; Hua, Xuanwen; Han, Keyi; Loughrey, David; Hatit, Marine Z C; Del Cid, Ada; Ni, Huanzhen; Shajii, Aram; Li, Andrea; Muralidharan, Abinaya; Peck, Hannah E; Tiegreen, Karen E; Jia, Shu; Santangelo, Philip J; Dahlman, James E | Proceedings of the National Academy of Sciences of the United States of America | 38437539 | 2024 Mar 12 |
Targeted nonviral delivery of genome editors in vivo. | Tsuchida, Connor A; Wasko, Kevin M; Hamilton, Jennifer R; Doudna, Jennifer A | Proceedings of the National Academy of Sciences of the United States of America | 38437567 | 2024 Mar 12 |
A humanized mouse model for adeno-associated viral gene therapy. | Barzi, Mercedes; Chen, Tong; Gonzalez, Trevor J; Pankowicz, Francis P; Oh, Seh Hoon; Streff, Helen L; Rosales, Alan; Ma, Yunhan; Collias, Sabrina; Woodfield, Sarah E; Diehl, Anna Mae; Vasudevan, Sanjeev A; Galvan, Thao N; Goss, John; Gersbach, Charles A; Bissig-Choisat, Beatrice; Asokan, Aravind; Bissig, Karl-Dimiter | Nature communications | 38438373 | 2024 Mar 04 |
Bacteriophage therapy for the treatment of Mycobacterium tuberculosis infections in humanized mice. | Yang, Fan; Labani-Motlagh, Alireza; Bohorquez, Jose Alejandro; Moreira, Josimar Dornelas; Ansari, Danish; Patel, Sahil; Spagnolo, Fabrizio; Florence, Jon; Vankayalapati, Abhinav; Sakai, Tsuyoshi; Sato, Osamu; Ikebe, Mitsuo; Vankayalapati, Ramakrishna; Dennehy, John J; Samten, Buka; Yi, Guohua | Communications biology | 38461214 | 2024 Mar 09 |
Hachiman is a genome integrity sensor. | Tuck, Owen T; Adler, Benjamin A; Armbruster, Emily G; Lahiri, Arushi; Hu, Jason J; Zhou, Julia; Pogliano, Joe; Doudna, Jennifer A | bioRxiv : the preprint server for biology | 38464307 | 2024 Feb 29 |
Long-term efficacy and safety of cardiac genome editing for catecholaminergic polymorphic ventricular tachycardia. | Moore, Oliver M; Aguilar-Sanchez, Yuriana; Lahiri, Satadru K; Hulsurkar, Mohit M; Alberto Navarro-Garcia, J; Word, Tarah A; Keefe, Joshua A; Barazi, Dean; Munivez, Elda M; Moore, Charles T; Parthasarathy, Vaidya; Davidson, Jaysón; Lagor, William R; Park, So Hyun; Bao, Gang; Miyake, Christina Y; Wehrens, Xander H T | The journal of cardiovascular aging | 38464671 | 2024 Jan |
A Novel Humanized Mouse Model for HIV and Tuberculosis Co-infection Studies. | Bohórquez, José Alejandro; Adduri, Sitaramaraju; Ansari, Danish; John, Sahana; Florence, Jon; Adejare, Omoyeni; Singh, Gaurav; Konduru, Nagarjun; Jagannath, Chinnaswamy; Yi, Guohua | bioRxiv : the preprint server for biology | 38496484 | 2024 Mar 07 |
Precision Enhancement of CAR-NK Cells through Non-Viral Engineering and Highly Multiplexed Base Editing. | Wang, Minjing; Krueger, Joshua B; Gilkey, Alexandria K; Stelljes, Erin M; Kluesner, Mitchell G; Pomeroy, Emily J; Skeate, Joseph G; Slipek, Nicholas J; Lahr, Walker S; Vázquez, Patricia N Claudio; Zhao, Yueting; Eaton, Ella J; Laoharawee, Kanut; Webber, Beau R; Moriarity, Branden S | bioRxiv : the preprint server for biology | 38496503 | 2024 Mar 08 |
Activation of the imprinted Prader-Willi Syndrome locus by CRISPR-based epigenome editing. | Rohm, Dahlia; Black, Joshua B; McCutcheon, Sean R; Barrera, Alejandro; Morone, Daniel J; Nuttle, Xander; de Esch, Celine E; Tai, Derek J C; Talkowski, Michael E; Iglesias, Nahid; Gersbach, Charles A | bioRxiv : the preprint server for biology | 38496583 | 2024 Mar 04 |
Adeno-associated virus as a delivery vector for gene therapy of human diseases. | Wang, Jiang-Hui; Gessler, Dominic J; Zhan, Wei; Gallagher, Thomas L; Gao, Guangping | Signal transduction and targeted therapy | 38565561 | 2024 Apr 03 |