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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