GENE-THERAPY PROSPECTS FOR DUCHENNE MUSCULAR-DYSTROPHY

被引：6

作者：

CLEMENS, PR ^{[1
]}

CASKEY, CT ^{[1
]}

机构：

[1] HOWARD HUGHES MED INST,HOUSTON,TX 77030

来源：

EUROPEAN NEUROLOGY | 1994年 / 34卷 / 04期

关键词：

DUCHENNE MUSCULAR DYSTROPHY; DYSTROPHIN; GENE TRANSFER THERAPY; VIRAL VECTOR;

D O I：

10.1159/000117035

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

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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页码：181 / 185

页数：5

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