Induced pluripotent stem cells from Huntington's disease patients:a promising approach to define and correct disease-related alterations  

作　　者：Azra Fatima Ricardo Gutiérrez-Garcia David Vilchez 

机构地区：[1]Institute for Genetics and Cologne Excellence Cluster for Cellular Stress Responses in Aging-Associated Diseases(CECAD),University of Cologne

出　　处：《Neural Regeneration Research》2019年第5期769-770,共2页中国神经再生研究（英文版）

基　　金：supported by the Else Kröner-Fresenius-Stiftung(2015_A118)

摘　　要：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.

关 键 词：ADULT SOMATIC CELLS skin or blood CELLS Huntington’s DISEASE PATIENTS 

分 类 号：R[医药卫生]