In FY15 the iPSC Core has collaborated and supported 28 NIH PIs and 3 extramural groups in iPSC-related research. We generated >400 iPSC clones from 100 human fibroblast and blood samples. We have developed and published semi-high-throughput fibroblast reprogramming method using latest non-integrating Sendai Virus (SeV) technology that allows efficient generation of iPSCs from up to 24 fibroblast samples simultaneously. We have developed robust SeV-mediated reprogramming protocol for blood samples from <4ml of whole blood. We have broad expertise on genetic manipulation in pluripotent stem cells. We have optimized designer nuclease (zinc finger nuclease, TALEN, CRISPR-Cas9)-mediated gene-editing application in human iPSCs, and generated knockout iPSC lines for 8 different genes. We have also used safe harbor designer nucleases to targeted integrate several transgenes and reporter genes in the safe harbor loci. These genetically modified iPSC lines are being used as isogenic and reporter lines to model disease and study differentiation and transplantation. We have developed and optimized several differentiation protocols relevant to cardiac and hematopoietic development to support NHLBI investigators' research. We have provided 5 validated control iPSC lines and various validated iPSC culture reagents to NIH investigators. We provided individual or group training of iPSC culture to 20 people from NHLBI, NCI, NEI, NICHD, NIAMS, NHGRI, NIA, NIMH, FDA. We also trained 10 people from NHLBI, NEI, NIDCR, NIAMS, and FDA to learn gene editing. We participated teaching at FAES course on cardiac differentiation. We presented posters at Keystone Symposium and NIH research festivals. We co-authored 9 papers in FY15. We continued to use iLab system to document and manage the Core services, and were able to generate the target revenue for FY15.

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Lin, Yongshun; Liu, Huimin; Klein, Michael et al. (2018) Efficient differentiation of cardiomyocytes and generation of calcium-sensor reporter lines from nonhuman primate iPSCs. Sci Rep 8:5907
Sima, Ni; Li, Rong; Huang, Wei et al. (2018) Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses. Orphanet J Rare Dis 13:54
Li, Pingjuan; Marino, Michael P; Zou, Jizhong et al. (2018) Efficiency and Specificity of Targeted Integration Mediated by the Adeno-Associated Virus Serotype 2 Rep 78 Protein. Hum Gene Ther Methods 29:135-145
Lin, Yongshun; Linask, Kaari L; Mallon, Barbara et al. (2017) Heparin Promotes Cardiac Differentiation of Human Pluripotent Stem Cells in Chemically Defined Albumin-Free Medium, Enabling Consistent Manufacture of Cardiomyocytes. Stem Cells Transl Med 6:527-538
Yada, Ravi Chandra; Ostrominski, John W; Tunc, Ilker et al. (2017) CRISPR/Cas9-Based Safe-Harbor Gene Editing in Rhesus iPSCs. Curr Protoc Stem Cell Biol 43:5A.11.1-5A.11.14
Hong, So Gun; Yada, Ravi Chandra; Choi, Kyujoo et al. (2017) Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ Layers. Mol Ther 25:44-53
Aguisanda, Francis; Yeh, Charles D; Chen, Catherine Z et al. (2017) Neural stem cells for disease modeling of Wolman disease and evaluation of therapeutics. Orphanet J Rare Dis 12:120
Chen, Guokai; Rao, Mahendra (2017) Derivation of Human-Induced Pluripotent Stem Cells in Chemically Defined Medium. Methods Mol Biol 1590:131-137
Han, Kim; Hassanzadeh, Shahin; Singh, Komudi et al. (2017) Parkin regulation of CHOP modulates susceptibility to cardiac endoplasmic reticulum stress. Sci Rep 7:2093
Sweeney, Colin L; Zou, Jizhong; Choi, Uimook et al. (2017) Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction. Mol Ther 25:321-330

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