Work

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

Year: 2003

Type: article

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.

Source: FEBS Letters

DOI: 10.1016/s0014-5793(03)00904-9

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

Institutions: Charing Cross Hospital, Imperial College London, Queen Elizabeth II Medical Centre

Language: English

Topic: RNA Interference and Gene Delivery

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: bronze

Funders: Muscular Dystrophy Association, Strong, Muscular Dystrophy UK, National Health and Medical Research Council