AAV

2024 MDA Summit Highlights Safety & Innovation in Gene Therapy

The 2024 Muscular Dystrophy Association Summit brought together international experts to discuss the scientific, operational, and ethical challenges of bringing gene therapies safely to patients. The meeting marks a major milestone in accelerating progress for neuromuscular diseases....
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Toward an AAV Gene Therapy to Treat Muscular Dystrophies

New innovations in AAV vector engineering — including dual-vector methods and enhanced tissue targeting — are opening doors for treating muscular dystrophies once considered untreatable....
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