The NHLBI iPSC Core Facility started service in May 2011. The lab temporarily operated in 10/6N240 till relocation to 10/5N214 in May 2012. The major activities in the lab include lab set up, training, collaboration with outside groups, iPSC derivation for the Institute and standard iPSCs for other intramural institutes. (iPSC: induced Pluripotent Stem Cells) A. Lab Set up and Services 1. Equipment set up. The lab was opened for consultation in May 2011 and we started to purchase essential equipment from May till August 2011. 2. The current lab site 6N240 was made operational for research for research in May 2011, the initial renovation was finished by the end of July, and the lab was functional in mid August 2011. 3. The initial iPSC derivation was started in Dr. Boehms lab in July, and later moved to 6N240. We have generated more than 124 lines from 33 patient and control samples. 4. We set up the operational procedure to generate defined medium for iPSC maintenance and derivation. We have generated 200L medium. 5. We developed iPSC derivation procedures in defined medium with lentivirus, episomal and Sendai virus approaches. We also tested fibroblast, CD34+, peripheral blood cells, HUVEC and adipocyte. 6. In collaboration with NIH-CRM, we prepared three NIH control cell lines that will be distributed to researchers in the community as standard iPSC line. 7. We developed protocols to evaluate incubation conditions and quality control steps for new reagents. We also routinely screened our cell culture. 8. A standard protocol was set up to analyze stem cell nuclear and surface markers. We are now able to analyze multiple samples with multiple antibodies in a high throughput fashion. 9. In collaboration with the Transgenic Core, we currently have protocols to conduct teratoma formation assay on SCID mice;and teratoma is stained and analyzed by Pathology Core. B. Training activities We held 8 workshops on human ESC/iPSC culture techniques. We hosted 36 NHLBI researchers from 19 NHLBI labs and we also trained 13 researchers from other NIH institutes and outside groups. We also provided additional consultations to NIH research groups. C. Interaction with outside groups 1. Collaboration with NIH-CRM on control cell lines. 2. We are the founding member of the US Stem Cell Core Facility network. Invited Talks NIH-CRM, 01/17/2012 Defining Cell Culture Conditions for Translational Research: Starting from Human Pluripotent Stem Cells. NCI-Frederick, 01/26/2012, Optimizing Stem Cell Culture Conditions for Translational Research. Reviewer duty for peer-reviewed journals: PLOS One, Stem Cells   D. iPSC lines derived in the lab. We successfully derived >124 iPSCs from 33 patient or control samples using lentivirus, Sendai virus and episomal approach. These cells will be used for disease modeling and protocol development. E. Control cell lines for the NHLBI and NIH. We banked 9 cell lines as control lines for NIH researchers. These lines will be used to generate lineage tracing cells and can be used to standardize conditions and develop new protocols.

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Kaewkhaw, Rossukon; Swaroop, Manju; Homma, Kohei et al. (2016) Treatment Paradigms for Retinal and Macular Diseases Using 3-D Retina Cultures Derived From Human Reporter Pluripotent Stem Cell Lines. Invest Ophthalmol Vis Sci 57:ORSFl1-ORSFl11
Lin, Yongshun; Linask, Kaari L; Mallon, Barbara et al. (2016) Heparin Promotes Cardiac Differentiation of Human Pluripotent Stem Cells in Chemically Defined Albumin-Free Medium, Enabling Consistent Manufacture of Cardiomyocytes. Stem Cells Transl Med :
Hong, So Gun; Lin, Yongshun; Dunbar, Cynthia E et al. (2016) The Role of Nonhuman Primate Animal Models in the Clinical Development of Pluripotent Stem Cell Therapies. Mol Ther 24:1165-9
Qin, Haiying; Cho, Monica; Haso, Waleed et al. (2015) Eradication of B-ALL using chimeric antigen receptor-expressing T cells targeting the TSLPR oncoprotein. Blood 126:629-39
Cerbini, Trevor; Luo, Yongquan; Rao, Mahendra S et al. (2015) 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 :
Kaewkhaw, Rossukon; Kaya, Koray Dogan; Brooks, Matthew et al. (2015) Transcriptome Dynamics of Developing Photoreceptors in Three-Dimensional Retina Cultures Recapitulates Temporal Sequence of Human Cone and Rod Differentiation Revealing Cell Surface Markers and Gene Networks. Stem Cells 33:3504-18
Zou, Donghua; McSweeney, Colleen; Sebastian, Aswathy et al. (2015) A critical role of RBM8a in proliferation and differentiation of embryonic neural progenitors. Neural Dev 10:18
Cerbini, Trevor; Funahashi, Ray; Luo, Yongquan et al. (2015) 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
Merling, Randall K; Sweeney, Colin L; Chu, Jessica et al. (2015) An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous disease. Mol Ther 23:147-57
Efthymiou, Anastasia G; Chen, Guibin; Rao, Mahendra et al. (2014) Self-renewal and cell lineage differentiation strategies in human embryonic stem cells and induced pluripotent stem cells. Expert Opin Biol Ther 14:1333-44

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