Department of Neurology,  the Second Hospital of Hebei Medical University,  Shijiazhuang 050017,  China

doi:10.3969/j.issn.1004-583X.2019.03.004

Abstract

Abstract: Duchenne muscular dystrophy (DMD) is an Xlinked recessive disorder caused by dystrophin gene mutations. It is characterized by progressive symmetrical skeletal muscle weakness and atrophy, and easy to be misdiagnosed on account of hidden onset in children and various clinical manifestations, including motor and nonmotor symptoms. Progress in the development of DMD gene therapy has been well made over the past 20 years. Numerous of researches are focused on exon skipping, nonsense readthrough, adenoassociated virusmediated micro/mini dystrophin gene delivery and gene editing using the CRISPR/Cas9 system. These innovative therapies are becoming the best means to treat DMD.

Key words: muscular dystrophy, Duchenne, dystrophin；symptoms &, signs, genes

Dong Hui, Song Xueqin. Department of Neurology, the Second Hospital of Hebei Medical University, Shijiazhuang 050017, China[J]. Clinical Focus, doi: 10.3969/j.issn.1004-583X.2019.03.004.

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URL: https://huicui.hebmu.edu.cn/EN/10.3969/j.issn.1004-583X.2019.03.004

https://huicui.hebmu.edu.cn/EN/Y2019/V34/I3/212

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Department of Neurology, the Second Hospital of Hebei Medical University, Shijiazhuang 050017, China

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