Article
In vivo genome editing in mouse restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers.
Genome medicine - 12 Apr 2021
Chen Menglong, Shi Hui, Gou Shixue, Wang Xiaomin, Li Lei, Jin Qin, Wu Han, Zhang Huili, Li Yaqin, Wang Liang, Li Huan, Lin Jinfu, Guo Wenjing, Jiang Zhiwu, Yang Xiaoyu, Xu Anding, Zhu Yuling, Zhang Cheng, Lai Liangxue, Li Xiaoping
Abstract excerpt
BACKGROUND: Mutations in the DMD gene encoding dystrophin-a critical structural element in muscle cells-cause Duchenne muscular dystrophy (DMD), which is the most common fatal genetic disease. Clustered regularly interspaced short palindromic repeat (CRISPR)-mediated gene editing is a promising strategy for permanently curing DMD. METHODS: In this study, we developed a novel strategy for reframing DMD mutations...
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