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

Int J Exp Pathol. 2003 Aug;84(4):165-72. doi: 10.1046/j.1365-2613.2003.00354.x.

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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Disease Models, Animal*
  • Dogs
  • Genetic Therapy / methods
  • Humans
  • Mice
  • Mice, Inbred mdx
  • Muscular Dystrophy, Animal / etiology*
  • Muscular Dystrophy, Animal / therapy
  • Muscular Dystrophy, Duchenne / etiology*
  • Muscular Dystrophy, Duchenne / therapy