Abstract:

Adult somatic cells such as skin or blood cells from either health donors or patients can be reprogrammed into induced pluripotent stem cells (iPSCs). Given their unlimited self-renewal and differentiation capacities, iPSCs are an invaluable resource to generate terminally differentiated cells. Thus, iPSCs can facilitate the study of human diseases and drug screening, holding great promise for regenerative medicine. Another significant advantage of iPSC disease-modeling is that normal and mutant proteins are expressed at endogenous levels. In addition, subtle phenotypes and the effects of genetic background variations can be assessed by comparison between iPSC lines obtained from different patients and healthy donors as well as isogenic lines, in which disease-related mutations are corrected.