Pharmacological enhancement of KCC2 gene expression exerts therapeutic effects on human Rett syndrome neurons and Mecp2 mutant mice

作者：Xin Lian Tang, Jesse Drotar, Keji Li, Cullen D. Clairmont, A. Sophie Brumm, Austin Sullins, Hao Wu, Xiaoxiao Shawn Liu, Jinhua Wang, Nathanael S. Gray, Mriganka Sur, Rudolf Jaenisch · 发表于：Science Translational Medicine · 年份：2019 · DOI：10.1126/scitranslmed.aau0164 · 被引用次数：124 · 研究领域：Genetics and Neurodevelopmental Disorders、Autism Spectrum Disorder Research、Family and Disability Support Research

mutant mice ameliorated disease-associated respiratory and locomotion phenotypes. The small-molecule compounds described in our study may have therapeutic effects not only in RTT but also in other neurological disorders involving dysregulation of KCC2.