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Stem Cell Lines (SCL)

RMP-generated induced pluripotent stem cell (iPSC) lines
Orphan and Rare disease iPSC lines

RMP-generated iPSC lines

During phase 1 of the RMP 1 clinical-grade, current Good Manufacturing Practice (cGMP) iPSC line and 14 research-grade iPSC lines were made available to the research community. These lines are distributed through RUCDR Infinite Biologics at Rutgers University. Please visit https://stemcells.nindsgenetics.org/ to request these lines and the frequently asked questions page for additional information, especially on the clinical-grade cell line. View an overview of the procedure for obtaining iPSCs from RUCDR Infinite Biologics.

Are you looking for human embryonic stem cell (hESC) lines? The NIH RMP has not supported the development of any hESC lines. For a list of available hESC lines eligible for NIH funding please visit the NIH Human Embryonic Stem Cell Registry.

Clinical Grade
Cell LineDescriptionTypeParental Line/Starting MaterialiPSC Reprogramming MethodStatus
LiPSC-GR1.1Current good manufacturing practices line (male)Clinical GradeCD34+ cord bloodEpisomal plasmidAvailable
LiPSC-GR1.1non-Current good manufacturing practices line (male)Research GradeCD34+ cord bloodEpisomal plasmidAvailable

 

Research Grade
Cell LineDescriptionTypeParental Line/Starting MaterialiPSC Reprogramming MethodStatus
NCRM-1NIH CRM control iPSC line (male)Control reference lineCD34+ cord bloodEpisomal plasmidAvailable
NCRM-2NIH CRM control iPSC line (female)Control reference lineCD34+ cord bloodEpisomal plasmidAvailable
NCRM-3NIH CRM control iPSC line (male)Control reference lineCD34+ cord bloodEpisomal plasmidAvailable
NCRM-4NIH CRM control iPSC line (female)Control reference lineCD34+ cord bloodEpisomal plasmidAvailable
NCRM-5NIH CRM control iPSC line (male)Control reference lineCD34+ cord bloodEpisomal plasmidAvailable
NCRM-6NIH CRM control iPSC line (female)Control reference lineCD34+ cord bloodEpisomal plasmidAvailable
ND1.4NIH CRM control iPSC lineControl reference lineFibroblast (ATCC)Episomal plasmidAvailable
ND2.0NIH CRM control iPSC lineControl reference lineFibroblast (ATCC)Episomal plasmidAvailable
CY2NIH CRM control iPSC lineControl reference lineBloodEpisomal plasmidAvailable
NCRM5-AS1-iCLHNNCRM-5 iPSCs targeted with NanoLuc-HaloTag at AAVS1 safe harborLuciferase reporter lineNCRM-5Episomal plasmidAvailable
NCRM5-C13-iCLHNNCRM-5 iPSCs targeted with NanoLuc-HaloTag at Chr. 13 safe harborLuciferase reporter lineNCRM-5Episomal plasmidAvailable
NCRM5-AS1-iCAGcGFPNCRM-5 iPSCs targeted with copGFP at AAVS1 safe harborGFP reporter lineNCRM-5Episomal plasmidAvailable
T21C1iPSC line from Down's syndrome patient (Trisomy 21)Trisomy 21 Down Syndrome linePatient fibroblasts (from Coriell)RetroviralAvailable
T21C5iPSC line from Down's syndrome patient in which trisomy 21 karyotype reverted to normalTrisomy 21 Down Syndrome linePatient fibroblasts (from Coriell)RetroviralAvailable

Publications that used RMP-generated iPSC lines from the above table

  1. Baghbaderani BA, Syama A, Sivapatham R, Pei Y, Mukherjee O, Fellner T, Zeng, Rao MS. Detailed Characterization of Human Induced Pluripotent Stem Cells Manufactured for Therapeutic Applications. Stem Cell Rev. 2016 Jun 10.
  2. Baghbaderani BA, Tian X, Neo BH, Burkall A, Dimezzo T, Sierra G, Zeng X, Warren K, Kovarcik DP, Fellner T, Rao MS. cGMP-Manufactured Human Induced Pluripotent Stem Cells Are Available for Pre-clinical and Clinical Applications. Stem Cell Reports. 5:647-659, 2015.
  3. Xue H, Wu J, Li S, Rao MS, Liu Y. Genetic Modification in Human Pluripotent Stem Cells by Homologous Recombination and CRISPR/Cas9 System. Methods Mol Biol. 1307:173-90, 2016.
  4. Efthymiou AG, Steiner J, Pavan WJ, Wincovitch S, Larson DM, Porter FD, Rao MS, Malik N. Rescue of an in vitro neuron phenotype identified in Niemann-Pick disease, type C1 induced pluripotent stem cell-derived neurons by modulating the WNT pathway and calcium signaling. Stem Cells Transl Med. 4:230-8, 2015.
  5. Li S, Xue H, Wu J, Rao MS, Kim DH, Deng W, Liu Y. Human Induced Pluripotent Stem Cell NEUROG2 Dual Knockin Reporter Lines Generated by the CRISPR/Cas9 System. Stem Cells Dev. 24:2925-42, 2015.
  6. Pei Y, Sierra G, Sivapatham R, Swistowski A, Rao MS, Zeng X. A platform for rapid generation of single and multiplexed reporters in human iPSC lines. Sci Rep. 5:9205, 2015.
  7. Cerbini T, Luo Y, Rao MS, Zou J. Transfection, selection, and colony-picking of human induced pluripotent stem cells TALEN-targeted with a GFP gene into the AAVS1 safe harbor. J Vis Exp. (96), 2015.
  8. Cerbini T, Funahashi R, Luo Y, Liu C, Park K, Rao M, Malik N, Zou J. Transcription activator-like effector nuclease (TALEN)-mediated CLYBL targeting enables enhanced transgene expression and one-step generation of dual reporter human induced pluripotent stem cell (iPSC) and neural stem cell (NSC) lines. PLoS One. 10:e0116032, 2015
  9. Luo Y, Liu C, Cerbini T, San H, Lin Y, Chen G, Rao MS, Zou J. Stable enhanced green fluorescent protein expression after differentiation and transplantation of reporter human induced pluripotent stem cells generated by AAVS1 transcription activator-like effector nucleases. Stem Cells Transl Med. 3:821-35, 2015.
  10. Malik N, Efthymiou AG, Mather K, Chester N, Wang X, Nath A, Rao MS, Steiner JP. Compounds with species and cell type specific toxicity identified in a 2000 compound drug screen of neural stem cells and rat mixed cortical neurons. Neurotoxicology. 45:192-200, 2014.
  11. Efthymiou A, Shaltouki A, Steiner JP, Jha B, Heman-Ackah SM, Swistowski A, Zeng X, Rao MS, Malik N. Functional screening assays with neurons generated from pluripotent stem cell-derived neural stem cells. J Biomol Screen. 19:32-43, 2014.
  12. Malik N, Wang X, Shah S, Efthymiou AG, Yan B, Heman-Ackah S, Zhan M, Rao M. Comparison of the gene expression profiles of human fetal cortical astrocytes with pluripotent stem cell derived neural stem cells identifies human astrocyte markers and signaling pathways and transcription factors active in human astrocytes. PLoS One. 9:e96139, 2014.
  13. Ou W, Li P, Reiser J. Targeting of herpes simplex virus 1 thymidine kinase gene sequences into the OCT4 locus of human induced pluripotent stem cells. PLoS One. 8:e81131, 2013.

Orphan and Rare disease iPSC lines

Phase 1 the RMP also supported NIH intramural investigators to develop iPSC lines from Orphan and Rare diseases and to help translate iPSC research into the clinic. These Orphan and Rare disease iPSC lines generated by NIH intramural investigators are available to the research community. Investigators interested in requesting an orphan and/or rare disease iPSC line should email the NIH intramural investigator who created the line (last column in chart below). Acknowledgement decisions are made between the requestor and the NIH investigator. Some of the iPSC lines were generated with materials from external clinicians and under different agreement types (IRB protocols and Material Transfer Agreements) and thus may carry use restrictions. Please email the NIH intramural investigator who created the line for additional information.

Orphan & Rare DiseaseiPSC LinesSexSourceNIH Intramural Investigator
Bilateral polydactylySCU-i1, SCU-i2, SCU-i3, SCU-i4FemalePrimary bone marrow stromal cellsPamela Robey
Bilateral polydactylySCU-i8, SCU-i9, SCU-i10FemalePrimary bone marrow stromal cellsPamela Robey
Fragile X syndromeHT-GC-15A, HT-GC-15B, HT-GC-15CMaleC10259 fibroblastsKaren Usdin
Fragile X syndromeHT-KL-13-1, HT-GC-13A, HT-GC-13BMaleC10700 fibroblastsKaren Usdin
Fragile X syndromeHT-KL-14-1, HT-KL-14-2, HT-KL-14-3MaleC10147 fibroblastsKaren Usdin
Fragile X syndromeFX1UT1, FX1UT2MaleGM05848 fibroblastsKaren Usdin
BEST Vitelliform Macular DegenerationBEST1 Clone H p9 Patient lineKapil Bharti
BEST Vitelliform Macular DegenerationBEST1 Clone I p9 Patient lineKapil Bharti
Adult-Onset Autosomal Dominant Retinal DegenerationClone 1A p8 Patient lineKapil Bharti
Adult-Onset Autosomal Dominant Retinal DegenerationClone A2 p5 Patient lineKapil Bharti
Adult-Onset Autosomal Dominant Retinal DegenerationControl 18E p9 Patient lineKapil Bharti
Adult-Onset Autosomal Dominant Retinal DegenerationControl 24L p9 Patient lineKapil Bharti
Leber's congenital amaurosis; Gene mutation: Cep290PEN1B, PEN1C Patient lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: Cep290PEN2A, PEN2D, PEN2F Patient lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: Cep290PEN3A, PEN3B Patient lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: Cep290PEN4A, PEN4C, PEN4E, PEN4F Patient lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: Cep290PEN5E, PEN5F Patient lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: Cep290NEI-001_NE, NEI-001_NF Patient lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: Cep290PEN6A, PEN6B Control lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: Cep290PEN7B, PEN7D Control lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: Cep290PEN8B, PEN8C, PEN8E, PEN8F Control lineAnand Swaroop
Joubert Syndrome; Gene mutation: Cep290Jou441-A, Jou441-B, Jou441-C, Jou441-D, Jou441-E, Jou441-F Patient lineAnand Swaroop
Joubert Syndrome; Gene mutation: Cep290Jou373-A, Jou373-C, Jou373-D, Jou373-F Patient lineAnand Swaroop
Joubert Syndrome; Gene mutation: Cep290Jou377-A, Jou377-C, Jou377-D, Jou377-E Control lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: CrxNEI001 Patient-A, NEI001 Patient-B, NEI001 Patient-C, NEI001 Patient-D Patient lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: CrxNEI002 Patient-A, NEI002 Patient-B Patient lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: CrxNEI001XX Control-A, NEI001XX Control-B, NEI001XX Control-C, NEI001XX Control-D Control lineAnand Swaroop
Leber's congenital amaurosis; Gene mutation: CrxNEI002XX Control-A, NEI002XX Control-B Control lineAnand Swaroop
Senior Loken; Gene mutation: Nphp5NEI-805-A, NEI-805-B Patient lineAnand Swaroop
Senior Loken; Gene mutation: Nphp5NEI-804-A, NEI-804-B Control lineAnand Swaroop
Autosomal Dominant Hyper IgE SyndromeND1A, ND1C, ND1F, ND1GMalePatient lineManfred Boehm
Autosomal Dominant Hyper IgE SyndromeND1B, ND1D, ND1EFemalePatient lineManfred Boehm
Arterial Calcification due to Deficiency of CD73ND2A, ND2B, ND2C, ND2DFemalePatient lineManfred Boehm
NEMO SyndromeND3A, ND3BMalePatient lineManfred Boehm
Adenosine Deaminase 2 DeficiencyND7A, ND7DMalePatient lineManfred Boehm
Adenosine Deaminase 2 DeficiencyND7B, ND7CFemalePatient lineManfred Boehm
Adenosine Deaminase 2 DeficiencyND7E, ND7F Patient lineManfred Boehm
Turner SyndromeND8A, ND8B, ND8CFemalePatient lineManfred Boehm
CCR5-Δ32 mutation (protects certain individuals from HIV)ND9AFemalePatient lineManfred Boehm
CCR5-Δ32 mutation (protects certain individuals from HIV)ND9B, ND9CMalePatient lineManfred Boehm
STING-associated Vasculopathy with onset in infancyND11AFemalePatient lineManfred Boehm
STING-associated Vasculopathy with onset in infancyND11B, ND11C, ND11D Patient lineManfred Boehm
STING-associated Vasculopathy with onset in infancy unaffected family memberNC11FemaleControl lineManfred Boehm
Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and LeukoencephalopathyND5MalePatient lineManfred Boehm
Generalized Arterial Calcification of InfancyND12AMalePatient lineManfred Boehm
Generalized Arterial Calcification of InfancyND12BFemalePatient lineManfred Boehm
Degos diseaseND13AMalePatient lineManfred Boehm
Degos diseaseND13B, ND13CFemalePatient lineManfred Boehm
Normal Control (no disease)NC1, NC1, NC2, NC5, NC6, NC7FemaleControl lineManfred Boehm
Normal Control (no disease)NC3, NC4, NC8, NC10MaleControl lineManfred Boehm
Normal Control (no disease)NC9 Control lineManfred Boehm
Spinal and bulbar muscular atrophy  PatientKenneth Fischbeck

This page last reviewed on January 17, 2024