Correction of human nonsense mutation via adenine base editing for Duchenne muscular dystrophy treatment in mouse.

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第一作者： Ming,Jin

第一单位： Department of Neurology and Institute of Neurology of First Affiliated Hospital, Institute of Neuroscience, Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou 350004, China.

作者： Ming,Jin ^[1] ; Jiajia,Lin ^[1] ; Haisen,Li ^[2] ; Zhifang,Li ^[3] ; Dong,Yang ^[4] ; Yin,Wang ^[2] ; Yuyang,Yu ^[2] ; Zhurui,Shao ^[2] ; Long,Chen ^[2] ; Zhiqiang,Wang ^[1] ; Yu,Zhang ^[1] ; Xiumei,Zhang ^[2] ; Ning,Wang ^[2] ; Chunlong,Xu ^[1] ; Hui,Yang ^[4] ; Wan-Jin,Chen ^[5] ; Guoling,Li ^[2]

作者单位： Department of Neurology and Institute of Neurology of First Affiliated Hospital, Institute of Neuroscience, Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou 350004, China. ^[1] HuidaGene Therapeutics Co., Ltd, Shanghai 200131, China. ^[2] School of Medicine, Wayne State University, Detroit, MI 48201, USA. ^[3] Lingang Laboratory, Shanghai 200031, China. ^[4] Shanghai Center for Brain Science and Brain-Inspired Technology, Shanghai 201602, China. ^[5] Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China. ^[6]

关键词 DMD MT: RNA/DNA Editing adenine base editing humanized mouse model nonsense mutation

DOI 10.1016/j.omtn.2024.102165

PMID 38571746

发布时间 2024-04-05

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