Duchenne muscular dystrophy: from gene to gene-ius therapies.

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular disorder caused by mutations in the dystrophin gene that result in the absence of functional dystrophin, leading to progressive muscle degeneration, loss of ambulation, respiratory failure, cardiomyopathy, and premature mortality. Despite advances in multidisciplinary supportive care, DMD remains an incurable disease associated with substantial...

Topics

• Humans
• Muscular Dystrophy, Duchenne
• Genetic Therapy
• Dystrophin
• Animals
• Gene Therapy Agents
• Mutation