title: 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.
authors.affiliation: MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford UK.
authors.forename: Rebecca J
authors.initials: RJ
authors.lastname: Fairclough
authors.forename: Akshay
authors.initials: A
authors.lastname: Bareja
authors.forename: Kay E
authors.initials: KE
authors.lastname: Davies
mesh_terms: D000818:Animals; D018389:Codon, Nonsense / Q000187:drug effects; D016189:Dystrophin / Q000235:genetics; D049028:Dystrophin-Associated Protein Complex / Q000235:genetics; D005091:Exons / Q000235:genetics; D016368:Frameshift Mutation; D015316:Genetic Therapy; D006801:Humans; D008297:Male; D051379:Mice; D018482:Muscle, Skeletal / Q000166:cytology / Q000254:growth & development; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000503:physiopathology / Q000628:therapy*; D032446:Myoblasts / Q000637:transplantation; D012508:Sarcolemma / Q000502:physiology; D015854:Up-Regulation; D049411:Utrophin / Q000235:genetics
chemical_list: D018389:Codon, Nonsense; D016189:Dystrophin; D049028:Dystrophin-Associated Protein Complex; D049411:Utrophin