Article
Multiplex epigenome editing of MECP2 to rescue Rett syndrome neurons.
Science translational medicine - 18 Jan 2023
Qian Junming, Guan Xiaonan, Xie Bing, Xu Chuanyun, Niu Jacqueline, Tang Xin, Li Charles H, Colecraft Henry M, Jaenisch Rudolf, Liu X Shawn
Abstract excerpt
Rett syndrome (RTT) is an X-linked neurodevelopmental disorder caused by loss-of-function heterozygous mutations of methyl CpG-binding protein 2 (MECP2) on the X chromosome in young females. Reactivation of the silent wild-type MECP2 allele from the inactive X chromosome (Xi) represents a promising therapeutic opportunity for female patients with RTT. Here, we applied a multiplex epigenome editing approach to...
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