Abstract | PURPOSE OF REVIEW: The search for a cure for Duchenne muscular dystrophy and other muscular dystrophies is progressing rapidly despite significant challenges posed by some genes and the complexity of targeting all skeletal muscles. This review focuses on three areas in which experimental clinical studies are in progress. RECENT FINDINGS: SUMMARY: These studies should provide proof of principle of these experimental approaches in humans. It is likely that further refinement will be required before extending their use to the wider community of patients with muscular dystrophy. In the meantime it is important to prepare accurate registries of molecularly characterized patients to facilitate their recruitment in future studies.
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Authors | Francesco Muntoni, Dominic Wells |
Journal | Current opinion in neurology
(Curr Opin Neurol)
Vol. 20
Issue 5
Pg. 590-4
(Oct 2007)
ISSN: 1350-7540 [Print] England |
PMID | 17885450
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)
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Chemical References |
- Dystrophin
- Oligonucleotides, Antisense
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Topics |
- Animals
- Dystrophin
(genetics)
- Genetic Predisposition to Disease
(genetics)
- Genetic Therapy
(methods, trends)
- Genetic Vectors
(genetics)
- Humans
- Muscular Dystrophies
(genetics, physiopathology, therapy)
- Muscular Dystrophy, Duchenne
(genetics, physiopathology, therapy)
- Oligonucleotides, Antisense
(genetics)
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