Gene Editing to the Rescue:Reversal of Social Deficits Associated with MECP2 Duplication  

作　　者：Zi-Xian Yu Dan-Yang Wang Xiao-Hong Xu 

机构地区：[1]Institute of Neuroscience,State Key Laboratory of Neuroscience,Chinese Academy of Sciences Center for Excellence in Brain Science and Intelligence Technology,Chinese Academy of Sciences,Shanghai 200031,China [2]University of the Chinese Academy of Sciences,Beijing 100049,China [3]Shanghai Center for Brain Science and Brain-Inspired Intelligence Technology,Shanghai 200031,China

出　　处：《Neuroscience Bulletin》2020年第6期567-569,共3页神经科学通报（英文版）

摘　　要：Methyl-CpG-binding protein 2 (MeCP2) is broadly recognized as the genetic cause of Rett Syndrome (RTT),a devastating neurodevelopmental disorder with the progressive loss in motor skills and speech that is found almost exclusively in young girls [1,2].Over 95%of RTT patients carry loss-of-function mutations in the X-linked MECP2gene,while the same mutations in males largely lead to infantile death.

关 键 词：MECP2 MECP2 RESCUE 

分 类 号：R749.94[医药卫生—神经病学与精神病学]