Gene therapy of muscular dystrophy

被引：85

作者：

Chamberlain, JS ^{[1
]}

机构：

[1] Univ Washington, Sch Med, Dept Neurol, Seattle, WA 98195 USA

来源：

HUMAN MOLECULAR GENETICS | 2002年 / 11卷 / 20期

基金：

美国国家卫生研究院;

关键词：

D O I：

10.1093/hmg/11.20.2355

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Development of gene therapy for the muscular dystrophies represents a daunting challenge requiring significant advances in our knowledge of the defective genes, muscle promoters, viral vectors, immune system surveillance and methods for systemic delivery of vectors. However, tremendous progress has been made in developing improved viral vectors and avoiding immune reactions against gene transfer. This review summarizes recent progress and highlights problems that must be solved before an effective treatment is available.

引用

页码：2355 / 2362

页数：8

共 115 条

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