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Animal models for muscular dystrophy : valuable tools for the development of therapies
Journal article   Open access   Peer reviewed

Animal models for muscular dystrophy : valuable tools for the development of therapies

Valérie ALLAMAND and Kevin P CAMPBELL
Human molecular genetics, Vol.9(16), pp.2459-2467
2000
DOI: 10.1093/hmg/9.16.2459
PMID: 11005802
url
https://doi.org/10.1093/hmg/9.16.2459View
Published (Version of record) Open Access

Abstract

Since the identification of dystrophin as the causative factor in Duchenne muscular dystrophy, an increasing amount of information on the molecular basis of muscular dystrophies has facilitated the division of these heterogeneous disorders into distinct groups. As more light is being shed on the genes and proteins involved in muscular dystrophy, diagnosis of patients has improved enormously. In addition to naturally occurring animal models, a number of genetically engineered murine models for muscular dystrophy have been generated. These animal models have provided valuable clues to the understanding of the pathogenesis of these disorders. Furthermore, as therapeutic approaches are being developed, mutant animals represent good models in which they can be tested. The present review focuses on the recent advancements of gene transfer-based strategies, with a special emphasis on animal models for Duchenne and limb-girdle muscular dystrophies.
Biotechnology Gene Therapy Fundamental and applied biological sciences. Psychology Classical genetics, quantitative genetics, hybrids Vertebrata Animals Health. Pharmaceutical industry Industrial applications and implications. Economical aspects Biological and medical sciences Genetics of eukaryotes. Biological and molecular evolution

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