Journal article
Splicing intervention for Duchenne muscular dystrophy
Current Opinion in Pharmacology, Vol.5(5), pp.529-534
2005
Abstract
The manipulation of pre-mRNA to alter gene transcript splicing patterns offers considerable potential for many genetic disorders. In particular, the targeted removal of one or more exons from a gene transcript can skip over, or compensate for, disease-causing mutations. Duchenne muscular dystrophy (DMD), the most common and severe form of muscular dystrophy, is one such disorder that could benefit from this strategy. Splicing modulation can convert a DMD phenotype into the less severe allelic Becker-like phenotype. Recent studies using antisense oligonucleotide-targeted exon skipping to induce near normal dystrophin in vivo in animal models, and in vitro in DMD cell lines, highlight the promise of this approach. On the basis of these successes, human clinical trials could be realized in the near future.
Details
- Title
- Splicing intervention for Duchenne muscular dystrophy
- Authors/Creators
- G. McClorey (Author/Creator)S. Fletcher (Author/Creator)S. Wilton (Author/Creator)
- Publication Details
- Current Opinion in Pharmacology, Vol.5(5), pp.529-534
- Publisher
- Elsevier
- Identifiers
- 991005542044407891
- Copyright
- 2005 Elsevier Ltd
- Murdoch Affiliation
- Murdoch University
- Language
- English
- Resource Type
- Journal article
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Source: InCites
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- Collaboration types
- Domestic collaboration
- Citation topics
- 1 Clinical & Life Sciences
- 1.255 Musculoskeletal Disorders
- 1.255.628 Duchenne Muscular Dystrophy
- Web Of Science research areas
- Pharmacology & Pharmacy
- ESI research areas
- Pharmacology & Toxicology