Inside Precision Medicine July 18, 2024
A new gene therapy treatment for Duchenne muscular dystrophy show promise of arresting the decline of the muscles of those affected by this inherited genetic disease, and perhaps, in the future, repairing those muscles. The laboratory results not only show promise to treat patients with this severely debilitating and incurable disease, but the technological advancement researchers used to create it may facilitate the development of other therapies in the future.
The new method, which has had success on mouse models, was published in the 17 July 2024 issue of Nature. It uses a series of adeno-associated viral vectors or AAVs, which are tiny shuttles derived from a virus that is commonly used to deliver gene therapies into human cells. Instead...
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