Molecular therapies for Duchenne muscular dystrophy.

Abstract excerpt

Duchenne muscular dystrophy (DMD) is the most common inherited muscle disorder, which is characterized by progressive muscle wasting, ultimately resulting in the death of patients in their twenties or thirties. In DMD the dystrophin gene is mutated, which results in a deficiency of the muscle dystrophin. Although expression of dystrophin is a fundamental treatment for DMD, no effective treatment for DMD is...

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