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Gene Therapy Prospects for Duchenne Muscular Dystrophy

 

作者: Paula R. Clemens,   C. Thomas Caskey,  

 

期刊: European Neurology  （Karger Available online 1994）
卷期: Volume 34, issue 4  

页码: 181-185

 

ISSN:0014-3022

 

年代: 1994

 

DOI:10.1159/000117035

 

出版商: S. Karger AG

 

关键词: Duchenne muscular dystrophy;Dystrophin;Gene transfer therapy;Viral vector

 

数据来源: Karger

 

摘要:

Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disorder caused by mutations in the dystrophin gene. The lack of adequate therapy for this disease provides impetus for the development of gene therapy strategies. A recombinant dystrophin cDNA and animal models of the disease are available for this therapy development. Characterization of these reagents and current progress toward gene therapy for DMD will be described.

 

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