In FY16, we continue to improve and add new technologies and services related iPSC research: We generated >500 iPSC clones from 70 human fibroblast and 55 blood samples for 15 NIH DIR Laboratories, surpassing FY15s record while maintaining 100% success rate. We have optimized CRISPR-Cas9-mediated gene-editing in human iPSCs, so that we can offer gene knockout, gene correction and safe harbor gene knockin services. In FY16, we have completed 23 gene-editing projects that include 15 gene knockout projects, 5 gene correction projects and 3 safe harbor gene knockin projects, generating >50 gene-edited lines for 7 NIH DIR Laboratories. These genetically modified iPSC lines are being used as isogenic and reporter lines to model human disease and study stem cell differentiation and transplantation. We have developed chemically defined media and a rapid 7-day protocol to differentiate human ESC/iPSC into >90% pure human cardiomyocytes (CM). In FY16, we provided iPSC-CM differentiation media kit and custom iPSC-CM differentiation services/support to 6 NHLBI and NCATS DIR Laboratories. We continue to provide validated control iPSC lines and various validated iPSC culture reagents to NIH investigators. We continue to use iLab system to document and manage Core services, and were able to recover 20% of our total budget . In FY16 the iPSC Core has collaborated with 16 NIH DIR Laboratories (12 of them are from NHLBI), and 3 extramural groups in iPSC-related research. Our research projects include studies of cardiovascular, hematopoietic, neurological, kidney, and metabolic diseases. We have used patient-derived iPSCs, gene-edited isogenic iPSCs, and iPSC-derived cardiomycocytes to elucidate transcriptional and epigenetic regulation of gene expression and to investigate how specific genetic mutations caused disease-specific phenotypes. We also established nonhuman primate iPSC lines which were further genetically engineered to express specific cell surface markers and then differentiated into cardiomyocytes. We provided individual or group training of iPSC culture, gene editing and cardiomyocyte differentiation. We participated teaching at FAES course on iPSC generation and cardiac differentiation. We presented posters at NHLBI DIR Research Festivals, NIH Summer Research Program Poster Day, and annual ISSCR meeting. We co-authored 3 papers in FY16.

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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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