Therapy of Genetic Disorders: Novel Therapies for Duchenne Muscular Dystrophy

被引：1
作者：
Seto J.T. [1 ]
Bengtsson N.E. [1 ]
Chamberlain J.S. [1 ]
机构：
[1] Department of Neurology, University of Washington, 1959 NE Pacific St, Seattle, 98195-7720, WA
来源：
Current Pediatrics Reports | 2014年 / 2卷 / 2期
基金：
美国国家卫生研究院; 英国医学研究理事会;
关键词：
Adeno-associated viral vectors; Antisense oligonucleotide; Duchenne muscular dystrophy; Dystrophin; Gene therapy; Inducible pluripotent stem cell; Mesoangioblast; Myoblast; Satellite cell;
D O I：
10.1007/s40124-014-0044-x
中图分类号：
学科分类号：
摘要：
Duchenne muscular dystrophy 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. © 2014, Springer Science + Business Media New York.
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收藏
页码：102 / 112
页数：10
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