Myopathy Press Releases

One of the biggest challenges in developing useful gene therapy is finding a way to get the beneficial gene into enough cells of the body to effectively treat the disease. Now, researchers have shown in rodents that a virus called adeno-associated virus 8 (AAV8) can effectively deliver a gene to all the skeletal muscles of the body. If it works the same way in humans, this virus-based approach may allow the first effective gene therapy for muscular dystrophy (MD) and similar diseases.

Researchers have revealed what may be a totally new cause for muscular dystrophy (MD). A recent study shows that a protein defective in two types of late-onset MD plays a critical role in the normal repair of muscles.
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