Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

被引：168

作者：

Fairclough, Rebecca J. ^{[1
,2
]}

Wood, Matthew J. ^{[1
]}

Davies, Kay E. ^{[1
,2
]}

机构：

[1] Univ Oxford, Dept Physiol Anat & Genet, Oxford OX1 3PT, England

[2] Univ Oxford, Dept Physiol Anat & Genet, MRC Funct Genom Unit, Oxford OX1 3PT, England

来源：

NATURE REVIEWS GENETICS | 2013年 / 14卷 / 06期

基金：

英国惠康基金; 英国医学研究理事会;

关键词：

MOUSE MODEL; MDX MOUSE; EXPRESSION; UTROPHIN; MUSCLE; RESTORATION; PGC-1-ALPHA; STRATEGIES; SARCOLEMMA; PATHOLOGY;

D O I：

10.1038/nrg3460

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Duchenne muscular dystrophy (DMD) is a devastating progressive disease for which there is currently no effective treatment except palliative therapy. There are several promising genetic approaches, including viral delivery of the missing dystrophin gene, read-through of translation stop codons, exon skipping to restore the reading frame and increased expression of the compensatory utrophin gene. The lessons learned from these approaches will be applicable to many other disorders.

引用

页码：373 / 378

页数：6

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