Department of Neurology, The University of Washington School of Medicine, Seattle, Washington 98105, USA.
Muscle Nerve. 2013 May;47(5):649-63. doi: 10.1002/mus.23738. Epub 2013 Mar 29.
Duchenne muscular dystrophy (DMD) is a devastating muscle disorder that affects 1 in 3,500 boys. Despite years of research and considerable progress in understanding the molecular mechanism of the disease and advancement of therapeutic approaches, there is no cure for DMD. The current treatment options are limited to physiotherapy and corticosteroids, and although they provide a substantial improvement in affected children, they only slow the course of the disorder. On a more optimistic note, more recent approaches either significantly alleviate or eliminate muscular dystrophy in murine and canine models of DMD and importantly, many of them are being tested in early phase human clinical trials. This review summarizes advancements that have been made in viral and nonviral gene therapy as well as stem cell therapy for DMD with a focus on the replacement and repair of the affected dystrophin gene.
杜氏肌营养不良症（DMD）是一种严重的肌肉疾病，每 3500 名男孩中就有 1 名患病。尽管多年来在了解疾病的分子机制和推进治疗方法方面取得了相当大的进展，但 DMD 仍然无法治愈。目前的治疗选择仅限于物理疗法和皮质类固醇，尽管它们为患病儿童提供了实质性的改善，但只能减缓疾病的进程。更乐观的是，最近的一些方法在 DMD 的鼠类和犬类模型中显著减轻或消除了肌肉营养不良，重要的是，其中许多方法正在早期人体临床试验中进行测试。本综述总结了在病毒和非病毒基因治疗以及 DMD 干细胞治疗方面的进展，重点是对受影响的肌营养不良蛋白基因的替代和修复。
