摘要：

近年来国外关于 Duchenne 型肌营养不良症基因治疗的临床试验取得了一定成果，疗效和安全性良好。我国 Duchenne 型肌营养不良症病例数较多，目前已纳入首批罕见病目录，可以预测基因治疗将很快进入我国。临床医师、患儿及其家属、政府相关部门、慈善基金会、媒体均应做好相应的准备，这将使我国 Duchenne型肌营养不良症患儿获益。

关键词: 肌营养不良症, 杜氏, 基因治疗, 综述

Abstract:

In recent years, the clinical trials of Duchenne muscular dystrophy (DMD) gene therapy in foreign countries have proved to be of certain curative effect and good safety. In China, DMD, with a large number of patients, has been introduced into the first rare diseases catalog, indicating that gene therapy will soon enter our country. It is a revolutionary treatment for DMD, and thus clinicians, patients and their families, relevant government departments, charity foundations and media shall prepare for the era of DMD gene therapy, which will benefit for DMD patients.

Key words: Muscular dystrophy, Duchenne, Genetic therapy, Review