Seto Jane T, Bengtsson Niclas E, Chamberlain Jeffrey S
Department of Neurology, 1959 NE Pacific St, University of Washington, Seattle, Washington 98195-7720, USA, Tel: 206-616-6645.
Curr Pediatr Rep. 2014 Jun 1;2(2):102-112. doi: 10.1007/s40124-014-0044-x.
Duchenne muscular dystrophy (DMD) is an inherited, progressive muscle wasting disorder caused by mutations in the dystrophin gene. An increasing variety of approaches are moving towards clinical testing that all aim to restore dystrophin production and to enhance or preserve muscle mass. Gene therapy methods are being developed to replace the defective dystrophin gene or induce dystrophin production from mutant genes. Stem cell approaches are being developed to replace lost muscle cells while also bringing in new dystrophin genes. This review summarizes recent progress in the field with an emphasis on clinical applications.
杜氏肌营养不良症（DMD）是一种由肌营养不良蛋白基因突变引起的遗传性进行性肌肉萎缩疾病。越来越多的方法正朝着临床试验迈进，所有这些方法都旨在恢复肌营养不良蛋白的产生，并增加或维持肌肉质量。正在开发基因治疗方法来替代有缺陷的肌营养不良蛋白基因，或诱导突变基因产生肌营养不良蛋白。正在开发干细胞方法来替代丢失的肌肉细胞，同时引入新的肌营养不良蛋白基因。本综述总结了该领域的最新进展，重点是临床应用。
