Wen Zhexing
Departments of Psychiatry and Behavioral Sciences, Cell Biology, and Neurology, Emory University School of Medicine, Atlanta, Georgia.
J Neurosci Res. 2017 May;95(5):1097-1109. doi: 10.1002/jnr.24031. Epub 2017 Feb 10.
Neurodevelopmental and psychiatric disorders, including autism spectrum disorder and schizophrenia, are complex and heterogeneous disorders that affect a large portion of the world's population. While the causes are still poorly understood, currently available treatments are limited; the development of rational therapeutics based on an understanding of the etiology and pathogenesis of the disease is imperative. The breakthrough technology of deriving induced pluripotent stem cells (iPSCs), reprogrammed from somatic cells of healthy subjects or patients, offers an unprecedented opportunity to recapitulate both normal and pathological development of human tissue, thereby opening up a new avenue for disease modeling and drug development in a more genetically tractable and disease-relevant system. Here, I review the recent progress in the use of human iPSCs for modeling neurodevelopmental and psychiatric disorders and developing novel therapeutic strategies, and discuss challenges in this rapidly moving field. © 2017 Wiley Periodicals, Inc.
神经发育障碍和精神疾病,包括自闭症谱系障碍和精神分裂症,是复杂的异质性疾病,影响着世界上很大一部分人口。虽然其病因仍知之甚少,但目前可用的治疗方法有限;基于对疾病病因和发病机制的理解来开发合理的治疗方法势在必行。从健康受试者或患者的体细胞重编程得到诱导多能干细胞(iPSC)这一突破性技术,为重现人类组织的正常和病理发育提供了前所未有的机会,从而在一个更易于进行基因操作且与疾病相关的系统中为疾病建模和药物开发开辟了一条新途径。在此,我综述了利用人类iPSC对神经发育障碍和精神疾病进行建模以及开发新型治疗策略的最新进展,并讨论了这个快速发展领域中的挑战。© 2017威利期刊公司