Progress in therapy for Duchenne muscular dystrophy.

Abstract:

:Duchenne muscular dystrophy is a devastating muscular dystrophy of childhood. Mutations in the dystrophin gene destroy the link between the internal muscle filaments and the extracellular matrix, resulting in severe muscle weakness and progressive muscle wasting. There is currently no cure and, whilst palliative treatment has improved, affected boys are normally confined to a wheelchair by 12 years of age and die from respiratory or cardiac complications in their twenties or thirties. Therapies currently being developed include mutation-specific treatments, DNA- and cell-based therapies, and drugs which aim to modulate cellular pathways or gene expression. This review aims to provide an overview of the different therapeutic approaches aimed at reconstructing the dystrophin-associated protein complex, including restoration of dystrophin expression and upregulation of the functional homologue, utrophin.

journal_name

Exp Physiol

journal_title

Experimental physiology

authors

Fairclough RJ,Bareja A,Davies KE

doi

10.1113/expphysiol.2010.053025

subject

Has Abstract

pub_date

2011-11-01 00:00:00

pages

1101-13

issue

11

eissn

0958-0670

issn

1469-445X

pii

expphysiol.2010.053025

journal_volume

96

pub_type

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