Gene therapy progress and prospects: Duchenne muscular dystrophy

被引:45
|
作者
Foster, K. [1 ]
Foster, H. [1 ]
Dickson, J. G. [1 ]
机构
[1] Univ London Royal Holloway & Bedford New Coll, Ctr Biomed Sci, Sch Biol Sci, Egham TW20 0EX, Surrey, England
关键词
Duchenne muscular dystrophy; systemic delivery; clinical trial;
D O I
10.1038/sj.gt.3302877
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder affecting 1/3500 male births. There is currently no effective treatment, but gene therapy approaches are offering viable avenues for treatment development. The last 10 years have seen the development of a number of strategies and tools for muscle gene therapy. However, the major hurdle has been the inability to deliver vectors at high enough efficiency via a systemic route. The last 2-3 years (reviewed here) have seen unrivalled progress in efficient systemic delivery of viral and non-viral gene transfer agents and antisense oligonucleotides. This progress, coupled with the successful completion of the first gene therapy clinical trial for DMD, has led to three more clinical trials planned for the immediate future.
引用
收藏
页码:1677 / 1685
页数:9
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