期刊
NATURE REVIEWS GENETICS
卷 14, 期 6, 页码 373-378出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nrg3460
关键词
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资金
- UK Medical Research Council
- Wellcome Trust
- UK Muscular Dystrophy Campaign
- US Muscular Dystrophy Association
- Action Duchenne
- Association Francaise Contre les Myopathies
- International Consortium on Exon Skipping
- Medical Research Council [MC_UU_12021/2, G0900887, G0801763] Funding Source: researchfish
- Rosetrees Trust [M164-F1] Funding Source: researchfish
- MRC [G0900887, MC_UU_12021/2, G0801763] Funding Source: UKRI
Duchenne muscular dystrophy (DMD) is a devastating progressive disease for which there is currently no effective treatment except palliative therapy. There are several promising genetic approaches, including viral delivery of the missing dystrophin gene, read-through of translation stop codons, exon skipping to restore the reading frame and increased expression of the compensatory utrophin gene. The lessons learned from these approaches will be applicable to many other disorders.
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