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

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.