Journal article
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
Nature Medicine, Vol.12(2), pp.175-177
2006
Abstract
For the majority of Duchenne muscular dystrophy (DMD) mutations, antisense oligonucleotide (AON)-mediated exon skipping has the potential to restore a functional protein. Here we show that weekly intravenous injections of morpholino phosphorodiamidate (morpholino) AONs induce expression of functional levels of dystrophin in body-wide skeletal muscles of the dystrophic mdx mouse, with resulting improvement in muscle function. Although the level of dystrophin expression achieved varies considerably between muscles, antisense therapy may provide a realistic hope for the treatment of a majority of individuals with DMD.
Details
- Title
- Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
- Authors/Creators
- J. Alter (Author/Creator) - Hammersmith HospitalF. Lou (Author/Creator) - University of HertfordshireA. Rabinowitz (Author/Creator) - Hammersmith HospitalH.F. Yin (Author/Creator) - Hammersmith HospitalJ. Rosenfeld (Author/Creator) - Carolinas Medical CenterS.D. Wilton (Author/Creator) - The University of Western AustraliaT.A. Partridge (Author/Creator) - Hammersmith HospitalQ.L. Lu (Author/Creator) - Carolinas Medical Center
- Publication Details
- Nature Medicine, Vol.12(2), pp.175-177
- Publisher
- Nature Publishing Group
- Identifiers
- 991005540922507891
- Copyright
- 2006 Nature Publishing Group
- Murdoch Affiliation
- Murdoch University
- Language
- English
- Resource Type
- Journal article
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Source: InCites
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- Collaboration types
- Domestic collaboration
- International collaboration
- Citation topics
- 1 Clinical & Life Sciences
- 1.255 Musculoskeletal Disorders
- 1.255.628 Duchenne Muscular Dystrophy
- Web Of Science research areas
- Biochemistry & Molecular Biology
- Cell Biology
- Medicine, Research & Experimental
- ESI research areas
- Molecular Biology & Genetics