Duchenne型肌营养不良症的治疗

doi:10.3969/j.issn.1004-583X.2021.02.015

摘要/Abstract

摘要：

Duchenne型肌营养不良症(Duchenne muscular dystrophy, DMD)是一种X连锁致死性遗传性肌病,由Dystrophin基因突变导致抗肌萎缩蛋白缺失所致。DMD尚缺乏有效的治疗方法,但随着对该病发病机制和病理变化过程的认识不断深入,其治疗选择越来越多。这些治疗方法旨在恢复Dystrophin蛋白表达或弥补Dystrophin蛋白缺失。

关键词: 肌营养不良, 杜氏, dystrophin蛋白, 基因治疗, 外显子跳跃

中图分类号:

R746.2

罗宏, 岑海媚, 罗彬, 张玉生, 张誉. Duchenne型肌营养不良症的治疗[J]. 临床荟萃, 2021, 36(2): 168-173.

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https://huicui.hebmu.edu.cn/CN/Y2021/V36/I2/168

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Duchenne型肌营养不良症的治疗

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