Duchenne's muscular dystrophy: animal models used to investigate pathogenesis and develop therapeutic strategies.

Abstract:

:Duchenne's muscular dystrophy (DMD) is a lethal childhood disease caused by mutations of the dystrophin gene, the protein product of which, dystrophin, has a vital role in maintaining muscle structure and function. Homologues of DMD have been identified in several animals including dogs, cats, mice, fish and invertebrates. The most notable of these are the extensively studied mdx mouse, a genetic and biochemical model of the human disease, and the muscular dystrophic Golden Retriever dog, which is the nearest pathological counterpart of DMD. These models have been used to explore potential therapeutic approaches along a number of avenues including gene replacement and cell transplantation strategies. High-throughput screening of pharmacological and genetic therapies could potentially be carried out in recently available smaller models such as zebrafish and Caenorhabditis elegans. It is possible that a successful treatment will eventually be identified through the integration of studies in multiple species differentially suited to addressing particular questions.

journal_name

Int J Exp Pathol

authors

Collins CA,Morgan JE

doi

10.1046/j.1365-2613.2003.00354.x

subject

Has Abstract

pub_date

2003-08-01 00:00:00

pages

165-72

issue

4

eissn

0959-9673

issn

1365-2613

pii

354

journal_volume

84

pub_type

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