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Stem Cell Lines

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.

 
RMP-generated iPSC lines - Clinical Grade
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

 

RMP-generated iPSC lines - Research Grade
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

  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 and Rare Disease iPSC lines
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 LORD Clone 1A p8   Patient line Kapil Bharti
Adult-Onset Autosomal Dominant Retinal Degeneration LORD Clone A2 p5   Patient line Kapil Bharti
Adult-Onset Autosomal Dominant Retinal Degeneration LORD Control 18E p9   Patient line Kapil Bharti
Adult-Onset Autosomal Dominant Retinal Degeneration LORD 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
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