Journal article
Rational design of antisense oligomers to induce dystrophin exon skipping
Molecular Therapy, Vol.17(8), pp.1418-1426
2009
Abstract
Duchenne muscular dystrophy (DMD), one of the most severe neuromuscular disorders of childhood, is caused by the absence of a functional dystrophin. Antisense oligomer (AO) induced exon skipping is being investigated to restore functional dystrophin expression in models of muscular dystrophy and DMD patients. One of the major challenges will be in the development of clinically relevant oligomers and exon skipping strategies to address many different mutations. Various models, including cell-free extracts, cells transfected with artificial constructs, or mice with a human transgene, have been proposed as tools to facilitate oligomer design. Despite strong sequence homology between the human and mouse dystrophin genes, directing an oligomer to the same motifs in both species does not always induce comparable exon skipping. We report substantially different levels of exon skipping induced in normal and dystrophic human myogenic cell lines and propose that animal models or artificial assay systems useful in initial studies may be of limited relevance in designing the most efficient compounds to induce targeted skipping of human dystrophin exons for therapeutic outcomes.
Details
- Title
- Rational design of antisense oligomers to induce dystrophin exon skipping
- Authors/Creators
- C. Mitrpant (Author/Creator) - The University of Western AustraliaA.M. Adams (Author/Creator) - The University of Western AustraliaP.L. Meloni (Author/Creator) - The University of Western AustraliaF. Muntoni (Author/Creator) - Institute of Child HealthS. Fletcher (Author/Creator) - The University of Western AustraliaS.D. Wilton (Author/Creator) - The University of Western Australia
- Publication Details
- Molecular Therapy, Vol.17(8), pp.1418-1426
- Publisher
- Nature Publishing Group
- Identifiers
- 991005542730807891
- Copyright
- 2009, The American Society of Gene & Cell Therapy
- Murdoch Affiliation
- Murdoch University
- Language
- English
- Resource Type
- Journal article
UN Sustainable Development Goals (SDGs)
This output has contributed to the advancement of the following goals:
Source: InCites
Metrics
27 Record Views
InCites Highlights
These are selected metrics from InCites Benchmarking & Analytics tool, related to this output
- 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
- Biotechnology & Applied Microbiology
- Genetics & Heredity
- Medicine, Research & Experimental
- ESI research areas
- Clinical Medicine