Regenerative Medicine Program (RMP)
Regenerative Medicine Program (RMP)
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.
| Cell Line | Description | Type | Parental Line/Starting Material | iPSC Reprogramming Method | Status |
|---|---|---|---|---|---|
| LiPSC-GR1.1 | Current good manufacturing practices line (male) | Clinical Grade | CD34+ cord blood | Episomal plasmid | Available |
| LiPSC-GR1.1 | non-Current good manufacturing practices line (male) | Research Grade | CD34+ cord blood | Episomal plasmid | Available |
| Cell Line | Description | Type | Parental Line/Starting Material | iPSC Reprogramming Method | Status |
|---|---|---|---|---|---|
| NCRM-1 | NIH CRM control iPSC line (male) | Control reference line | CD34+ cord blood | Episomal plasmid | Available |
| NCRM-2 | NIH CRM control iPSC line (female) | Control reference line | CD34+ cord blood | Episomal plasmid | Available |
| NCRM-3 | NIH CRM control iPSC line (male) | Control reference line | CD34+ cord blood | Episomal plasmid | Available |
| NCRM-4 | NIH CRM control iPSC line (female) | Control reference line | CD34+ cord blood | Episomal plasmid | Available |
| NCRM-5 | NIH CRM control iPSC line (male) | Control reference line | CD34+ cord blood | Episomal plasmid | Available |
| NCRM-6 | NIH CRM control iPSC line (female) | Control reference line | CD34+ cord blood | Episomal plasmid | Available |
| ND1.4 | NIH CRM control iPSC line | Control reference line | Fibroblast (ATCC) | Episomal plasmid | Available |
| ND2.0 | NIH CRM control iPSC line | Control reference line | Fibroblast (ATCC) | Episomal plasmid | Available |
| CY2 | NIH CRM control iPSC line | Control reference line | Blood | Episomal plasmid | Available |
| NCRM5-AS1-iCLHN | NCRM-5 iPSCs targeted with NanoLuc-HaloTag at AAVS1 safe harbor | Luciferase reporter line | NCRM-5 | Episomal plasmid | Available |
| NCRM5-C13-iCLHN | NCRM-5 iPSCs targeted with NanoLuc-HaloTag at Chr. 13 safe harbor | Luciferase reporter line | NCRM-5 | Episomal plasmid | Available |
| NCRM5-AS1-iCAGcGFP | NCRM-5 iPSCs targeted with copGFP at AAVS1 safe harbor | GFP reporter line | NCRM-5 | Episomal plasmid | Available |
| T21C1 | iPSC line from Down's syndrome patient (Trisomy 21) | Trisomy 21 Down Syndrome line | Patient fibroblasts (from Coriell) | Retroviral | Available |
| T21C5 | iPSC line from Down's syndrome patient in which trisomy 21 karyotype reverted to normal | Trisomy 21 Down Syndrome line | Patient fibroblasts (from Coriell) | Retroviral | Available |
Publications that used RMP-generated iPSC lines from the above table
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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
- 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.
- 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.
- 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.
- 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.
- 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 Disease | iPSC Lines | Sex | Source | NIH Intramural Investigator |
|---|---|---|---|---|
| Bilateral polydactyly | SCU-i1, SCU-i2, SCU-i3, SCU-i4 | Female | Primary bone marrow stromal cells | Pamela Robey |
| Bilateral polydactyly | SCU-i8, SCU-i9, SCU-i10 | Female | Primary bone marrow stromal cells | Pamela Robey |
| Fragile X syndrome | HT-GC-15A, HT-GC-15B, HT-GC-15C | Male | C10259 fibroblasts | Karen Usdin |
| Fragile X syndrome | HT-KL-13-1, HT-GC-13A, HT-GC-13B | Male | C10700 fibroblasts | Karen Usdin |
| Fragile X syndrome | HT-KL-14-1, HT-KL-14-2, HT-KL-14-3 | Male | C10147 fibroblasts | Karen Usdin |
| Fragile X syndrome | FX1UT1, FX1UT2 | Male | GM05848 fibroblasts | Karen Usdin |
| BEST Vitelliform Macular Degeneration | BEST1 Clone H p9 | Patient line | Kapil Bharti | |
| BEST Vitelliform Macular Degeneration | BEST1 Clone I p9 | Patient line | Kapil Bharti | |
| Adult-Onset Autosomal Dominant Retinal Degeneration | Clone 1A p8 | Patient line | Kapil Bharti | |
| Adult-Onset Autosomal Dominant Retinal Degeneration | Clone A2 p5 | Patient line | Kapil Bharti | |
| Adult-Onset Autosomal Dominant Retinal Degeneration | Control 18E p9 | Patient line | Kapil Bharti | |
| Adult-Onset Autosomal Dominant Retinal Degeneration | Control 24L p9 | Patient line | Kapil Bharti | |
| Leber's congenital amaurosis; Gene mutation: Cep290 | PEN1B, PEN1C | Patient line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Cep290 | PEN2A, PEN2D, PEN2F | Patient line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Cep290 | PEN3A, PEN3B | Patient line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Cep290 | PEN4A, PEN4C, PEN4E, PEN4F | Patient line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Cep290 | PEN5E, PEN5F | Patient line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Cep290 | NEI-001_NE, NEI-001_NF | Patient line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Cep290 | PEN6A, PEN6B | Control line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Cep290 | PEN7B, PEN7D | Control line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Cep290 | PEN8B, PEN8C, PEN8E, PEN8F | Control line | Anand Swaroop | |
| Joubert Syndrome; Gene mutation: Cep290 | Jou441-A, Jou441-B, Jou441-C, Jou441-D, Jou441-E, Jou441-F | Patient line | Anand Swaroop | |
| Joubert Syndrome; Gene mutation: Cep290 | Jou373-A, Jou373-C, Jou373-D, Jou373-F | Patient line | Anand Swaroop | |
| Joubert Syndrome; Gene mutation: Cep290 | Jou377-A, Jou377-C, Jou377-D, Jou377-E | Control line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Crx | NEI001 Patient-A, NEI001 Patient-B, NEI001 Patient-C, NEI001 Patient-D | Patient line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Crx | NEI002 Patient-A, NEI002 Patient-B | Patient line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Crx | NEI001XX Control-A, NEI001XX Control-B, NEI001XX Control-C, NEI001XX Control-D | Control line | Anand Swaroop | |
| Leber's congenital amaurosis; Gene mutation: Crx | NEI002XX Control-A, NEI002XX Control-B | Control line | Anand Swaroop | |
| Senior Loken; Gene mutation: Nphp5 | NEI-805-A, NEI-805-B | Patient line | Anand Swaroop | |
| Senior Loken; Gene mutation: Nphp5 | NEI-804-A, NEI-804-B | Control line | Anand Swaroop | |
| Autosomal Dominant Hyper IgE Syndrome | ND1A, ND1C, ND1F, ND1G | Male | Patient line | Manfred Boehm |
| Autosomal Dominant Hyper IgE Syndrome | ND1B, ND1D, ND1E | Female | Patient line | Manfred Boehm |
| Arterial Calcification due to Deficiency of CD73 | ND2A, ND2B, ND2C, ND2D | Female | Patient line | Manfred Boehm |
| NEMO Syndrome | ND3A, ND3B | Male | Patient line | Manfred Boehm |
| Adenosine Deaminase 2 Deficiency | ND7A, ND7D | Male | Patient line | Manfred Boehm |
| Adenosine Deaminase 2 Deficiency | ND7B, ND7C | Female | Patient line | Manfred Boehm |
| Adenosine Deaminase 2 Deficiency | ND7E, ND7F | Patient line | Manfred Boehm | |
| Turner Syndrome | ND8A, ND8B, ND8C | Female | Patient line | Manfred Boehm |
| CCR5-Δ32 mutation (protects certain individuals from HIV) | ND9A | Female | Patient line | Manfred Boehm |
| CCR5-Δ32 mutation (protects certain individuals from HIV) | ND9B, ND9C | Male | Patient line | Manfred Boehm |
| STING-associated Vasculopathy with onset in infancy | ND11A | Female | Patient line | Manfred Boehm |
| STING-associated Vasculopathy with onset in infancy | ND11B, ND11C, ND11D | Patient line | Manfred Boehm | |
| STING-associated Vasculopathy with onset in infancy unaffected family member | NC11 | Female | Control line | Manfred Boehm |
| Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy | ND5 | Male | Patient line | Manfred Boehm |
| Generalized Arterial Calcification of Infancy | ND12A | Male | Patient line | Manfred Boehm |
| Generalized Arterial Calcification of Infancy | ND12B | Female | Patient line | Manfred Boehm |
| Degos disease | ND13A | Male | Patient line | Manfred Boehm |
| Degos disease | ND13B, ND13C | Female | Patient line | Manfred Boehm |
| Normal Control (no disease) | NC1, NC1, NC2, NC5, NC6, NC7 | Female | Control line | Manfred Boehm |
| Normal Control (no disease) | NC3, NC4, NC8, NC10 | Male | Control line | Manfred Boehm |
| Normal Control (no disease) | NC9 | Control line | Manfred Boehm | |
| Spinal and bulbar muscular atrophy | Patient | Kenneth Fischbeck |
