The core generated more than 250 iPSC lines from over 80 patient samples for 7 NHLBI PIs, and multiple other NIH institutes, including NEI, NCAT, NIMH, NICHD, NINDS, NCI, NIAMS, and NHGRI. We developed a semi-high throughput and much more efficient platform to derivate iPSCs from 24 patient samples simultaneously. We transitioned to utilizing non-integrating Sendai vectors for reprogramming, as an available alternative to standard lentiviral vectors. We began optimizing reprogramming of alternative starting materials, specifically endothelial cells or peripheral blood mononuclear cells, because of investigator demand. Many investigators do not have access to skin fibroblasts as a starting cell population for reprogramming. We trained investigators to handle and maintain the lines in more than 18 individual and small group workshops. We directed two FAES courses on iPSCs and one on cardiac differentiation. We presented posters at a Gordon Conference and a Keystone Symposium. We developed and optimized several differentiation protocols of relevance to NHLBI investigators, including embryoid body differentiation and cardiomyocyte and hepatocyte differentiation. We set up in-house and tested TALEN assembly procedures for genomic engineering, and have begun to set up CRISPR/Cas9 genome engineering, which will be offered as a service in 2015. We continued to use iLab system to document and manage the Core services, and were able to generate the target revenue for fiscal year 2014.

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3
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2014
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U.S. National Heart Lung and Blood Inst
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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
Yada, Ravi Chandra; Hong, So Gun; Lin, Yongshun et al. (2017) Rhesus Macaque iPSC Generation and Maintenance. Curr Protoc Stem Cell Biol 41:4A.11.1-4A.11.13
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

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