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首页> 外文期刊>Human gene therapy. Clinical development >Dystrophin Gene Replacement and Gene Repair Therapy for Duchenne Muscular Dystrophy in 2016: An Interview
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Dystrophin Gene Replacement and Gene Repair Therapy for Duchenne Muscular Dystrophy in 2016: An Interview

机译：肌营养不良蛋白基因替换和基因修复疗法在2016年的杜兴氏肌营养不良症：访谈

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摘要

After years of relentless efforts, gene therapy has now begun to deliver its therapeutic promise in several diseases. A number of gene therapy products have received regulatory approval in Europe and Asia. Duchenne muscular dystrophy (DMD) is an X-linked inherited lethal muscle disease. It is caused by mutations in the dystrophin gene. Replacing and/or repairing the mutated dystrophin gene holds great promises to treated DMD at the genetic level. Last several years have evidenced significant developments in preclinical experimentations in murine and canine models of DMD. There has been a strong interest in moving these promising findings to clinical trials. In light of rapid progress in this field, the Parent Project Muscular Dystrophy (PPMD) recently interviewed me on the current status of DMD gene therapy and readiness for clinical trials. Here I summarized the interview with PPMD.
机译：经过多年的不懈努力，基因治疗现已开始在多种疾病中发挥其治疗前景。许多基因治疗产品已在欧洲和亚洲获得监管机构的批准。 Duchenne肌营养不良症（DMD）是X连锁遗传性致死性肌肉疾病。它是由肌营养不良蛋白基因的突变引起的。替换和/或修复突变的肌营养不良蛋白基因在基因水平上对治疗DMD具有广阔的前景。最近几年已证明在DMD的鼠科动物和犬科动物模型的临床前实验中取得了重大进展。将这些有前途的发现应用于临床试验引起了人们极大的兴趣。鉴于该领域的快速发展，“父母项目肌肉营养不良症”（PPMD）最近就DMD基因治疗的现状和临床试验准备情况采访了我。在这里，我总结了对PPMD的采访。

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