THE VALUE OF MAMMALIAN MODELS FOR DUCHENNE MUSCULAR DYSTROPHY IN DEVELOPING THERAPEUTIC STRATEGIES

被引:93
作者
Banks, Glen B. [1 ]
Chamberlain, Jeffrey S. [1 ]
机构
[1] Univ Washington, Dept Neurol, Seattle, WA 98195 USA
来源
MOUSE MODELS OF DEVELOPMENTAL GENETIC DISEASE | 2008年 / 84卷
基金
美国国家卫生研究院; 英国医学研究理事会;
关键词
D O I
10.1016/S0070-2153(08)00609-1
中图分类号
Q [生物科学];
学科分类号
07 ; 0710 ; 09 ;
摘要
Duchenne muscular dystrophy (DUD) is the most common form of muscular dystrophy. There is no effective treatment and patients typically die in approximately the third decade. DMD is an X-linked recessive disease caused by mutations in the dystrophin gene. There are three mammalian models of DMD that have been used to understand better the pathogenesis of disease and develop therapeutic strategies. The mdx mouse is the most widely used model of DMD that displays some features of muscle degeneration, but the pathogenesis of disease is comparatively mild. The severity of disease in mice lacking both dystrophin and utrophin is similar to DUD, but one has to account for the discrete functions of utrophin. Canine X-linked muscular dystrophy (cxmd) is the best representation of DUD, but the phenotype of the most widely used golden retriever (GRMD) model is variable, making functional endpoints difficult to ascertain. Although each mammalian model has its limitations, together they have been essential for the development of several treatment strategies for DMD that target dystrophin replacement, disease progression, and muscle regeneration.
引用
收藏
页码:431 / 453
页数:23
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