Induced dystrophin exon skipping in human muscle explants

G. McClorey; A.M. Fall; H.M. Moulton; P.L. Iversen; J.E. Rasko; M. Ryan; S. Fletcher; S.D. Wilton

doi:10.1016/j.nmd.2006.05.017

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Induced dystrophin exon skipping in human muscle explants

Journal article

Peer reviewed

Induced dystrophin exon skipping in human muscle explants

G. McClorey, A.M. Fall, H.M. Moulton, P.L. Iversen, J.E. Rasko, M. Ryan, S. Fletcher and S.D. Wilton

Neuromuscular Disorders, Vol.16(9-10), pp.583-590

2006

DOI: https://doi.org/10.1016/j.nmd.2006.05.017

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Abstract

Antisense oligonucleotide (AO) manipulation of pre-mRNA splicing of the dystrophin gene is showing promise in overcoming Duchenne muscular dystrophy (DMD)-causing mutations. To date, this approach has been limited to studies using animal models or cultured human muscle cells, and evidence that AOs can induce exon skipping in human muscle has yet to be shown. In this study, we used different AO analogues to induce exon skipping in muscle explants derived from normal and DMD human tissue. We propose that inducing exon skipping in human muscle explants is closer to in vivo conditions than cells in monolayer cultures, and may minimize the numbers of participants in Phase I clinical studies to demonstrate proof of principle of exon skipping in human muscle.

Details

Title: Induced dystrophin exon skipping in human muscle explants
Authors/Creators: G. McClorey (Author/Creator) - The University of Western Australia
A.M. Fall (Author/Creator) - The University of Western Australia
H.M. Moulton (Author/Creator) - Sarepta Therapeutics (United States)
P.L. Iversen (Author/Creator) - Sarepta Therapeutics (United States)
J.E. Rasko (Author/Creator) - Centenary Institute
M. Ryan (Author/Creator) - Children's Hospital at Westmead
S. Fletcher (Author/Creator) - The University of Western Australia
S.D. Wilton (Author/Creator) - The University of Western Australia
Publication Details: Neuromuscular Disorders, Vol.16(9-10), pp.583-590
Publisher: Elsevier BV
Identifiers: 991005542185407891
Copyright: 2006 Elsevier B.V
Murdoch Affiliation: Murdoch University
Language: English
Resource Type: Journal article

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Collaboration types: Industry collaboration; 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: Clinical Neurology; Neurosciences
ESI research areas: Neuroscience & Behavior