Induced pluripotent stem cells from Huntington's disease patients: a promising approach to define and correct disease-related alterations
Azra Fatima
Ricardo Gutiérrez-Garcia
David Vilchez
摘要：Adult somatic cells such as skin or blood cells from either health do-nors 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 ob-tained from different patients and healthy donors as well as isogenic lines, in which disease-related mutations are corrected.
机标关键词：
资助基金：the Else Kr?ner-Fresenius-Stiftung (2015_A118)
论文发表日期：2019-01-01
在线出版日期：2025-08-15（本平台首次上网日期，不代表文献的发表时间）
页数：2( 769-770 )