Journal
FEBS JOURNAL
Volume 280, Issue 17, Pages 4263-4280Publisher
WILEY
DOI: 10.1111/febs.12178
Keywords
cell therapy; gene therapy; genome editing; mRNA modifications; muscle regeneration; muscular dystrophy; stem cells
Categories
Funding
- UK Medical Research Council
- European Research Council
- European Community 7th Framework project Optistem
- Italian Duchenne Parent Project
- MRC [MR/J006785/1] Funding Source: UKRI
- Medical Research Council [MR/J006785/1] Funding Source: researchfish
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Muscular dystrophies are genetic disorders characterized by skeletal muscle wasting and weakness. Although there is no effective therapy, a number of experimental strategies have been developed over recent years and some of them are undergoing clinical investigation. In this review, we highlight recent developments and key challenges for strategies based upon gene replacement and gene/expression repair, including exon-skipping, vector-mediated gene therapy and cell therapy. Therapeutic strategies for different forms of muscular dystrophy are discussed, with an emphasis on Duchenne muscular dystrophy, given the severity and the relatively advanced status of clinical studies for this disease.
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