Clemens P R, Caskey C T
Institute for Molecular Genetics, Baylor College of Medicine, Houston, TX 77030.
Eur Neurol. 1994;34(4):181-5. doi: 10.1159/000117035.
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.
杜兴氏肌肉营养不良症（DMD）是一种由肌营养不良蛋白基因突变引起的毁灭性神经肌肉疾病。针对这种疾病缺乏足够的治疗方法，这为基因治疗策略的发展提供了动力。一种重组肌营养不良蛋白cDNA和该疾病的动物模型可用于这种治疗方法的开发。将描述这些试剂的特性以及目前针对DMD基因治疗的进展。
