Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle

K.E. Wells; S. Fletcher; C.J. Mann; S.D. Wilton; D.J. Wells

doi:10.1016/S0014-5793(03)00904-9

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Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle

Journal article

Peer reviewed

Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle

K.E. Wells, S. Fletcher, C.J. Mann, S.D. Wilton and D.J. Wells

FEBS Letters, Vol.552(2-3), pp.145-149

2003

DOI: https://doi.org/10.1016/S0014-5793(03)00904-9

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Abstract

The use of antisense oligonucleotides (AOs) to induce exon skipping leading to generation of an in-frame dystrophin protein product could be of benefit in around 70% of Duchenne muscular dystrophy patients. We describe the use of hyaluronidase enhanced electrotransfer to deliver uncomplexed 2′-O-methyl modified phosphorothioate AO to adult dystrophic mouse muscle, resulting in dystrophin expression in 20–30% of fibres in tibialis anterior muscle after a single injection. Although expression was transient, many of the corrected fibres initially showed levels of dystrophin expression well above the 20% of endogenous previously shown to be necessary for phenotypic correction of the dystrophic phenotype.

Details

Title: Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle
Authors/Creators: K.E. Wells (Author/Creator) - Charing Cross Hospital
S. Fletcher (Author/Creator) - Queen Elizabeth II Medical Centre
C.J. Mann (Author/Creator) - Queen Elizabeth II Medical Centre
S.D. Wilton (Author/Creator) - Queen Elizabeth II Medical Centre
D.J. Wells (Author/Creator) - Charing Cross Hospital
Publication Details: FEBS Letters, Vol.552(2-3), pp.145-149
Publisher: Elsevier B.V.
Identifiers: 991005543861107891
Copyright: N 2003 Federation of European Biochemical Societies
Murdoch Affiliation: Murdoch University
Language: English
Resource Type: Journal article

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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; Biophysics; Cell Biology
ESI research areas: Biology & Biochemistry