Stem Cells and Disease Modeling

Human pluripotent stem cells (hPSCs) offer a useful model system for understanding the genetic basis of human cardiovascular diseases. Unlike nonhuman animal models, hPSCs are often tightly genetically matched to patients with disease. Previously, only in unusual circumstances was it feasible to review primary tissues like heart muscle and blood vessels obtained directly from living patients, and even in such circumstances, the quantity of tissue was limited. hPSCs represent a replacement approach that gives an unprecedented opportunity to review human cells that are matched to the patients of interest. Because they will be expanded into very large numbers and differentiated into a spread of cell types that are relevant to cardiovascular diseases—including cardiomyocytes, vascular endothelial and smooth muscle cells, and hepatocytes—hPSCs can in theory provide a limitless source of fabric with which to dissect the molecular underpinnings of the patient’s disease process within and beyond the circulatory system.


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