Isogenic human pluripotent stem cell-based models of human disease mutationsIsogenic human pluripotent stem cell-based models of human disease mutations

‚ÄčOur TR01 project integrates human genetics, genome engineering, and stem cell biology to advance understanding of diabetes, dyslipidemia, and coronary artery disease. The image represents three recent technological advances that make our project possible: (a) next-generation genome sequencing of human clinical populations, (b) methods to rapidly engineer mutations of interest into the genome of human pluripotent stem cells (hPSCs) using TAL effector nucleases (TALENs), and (c) protocols to differentiate hPSCs into cell populations with characteristics of mature hepatocytes, adipocytes, and pancreatic beta cells.