Journal
NATURE REVIEWS GENETICS
Volume 14, Issue 6, Pages 373-378Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nrg3460
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Funding
- 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
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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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