An attempt of gene therapy in Duchenne muscular dystrophy: overexpression of utrophin in transgenic mdx mice.

Dystrophin, its functions and the consequences of its absence are briefly reviewed. The animal model of Duchenne myopathy, the mdx mouse, was used to over-express utrophin by transgenesis technology. A battery of functional tests, including mechanical responses (force development and resistance to imposed stretch), intracellular calcium homeostasis and metabolic reaction to muscle activity were applied to check the functional recovery obtained by over-expression of utrophin. For most parameters tested, recovery amounted to 80%, demonstrating that utrophin can very efficiently act as a surrogate for dystrophin.