In Vivo Genome Editing in Mouse Restores Dystrophin Expression in Duchenne Muscular Dystrophy Patient Muscle Fibers.

Menglong Chen,Hui Shi,Shixue Gou,Xiaomin Wang,Lei Li,Qin Jin,Han Wu,Huili Zhang,Yaqin Li, Liang Wang,Huan Li,Jinfu Lin,Wenjing Guo,Zhiwu Jiang,Xiaoyu Yang,Anding Xu,Yuling Zhu,Cheng Zhang,Liangxue Lai, Xiaoping Li

Genome medicine（2021）

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关键词
Duchenne muscular dystrophy,Gene editing,CRISPR,Cas9,CRISPR,Cas12a,Patient-derived xenograft model
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