A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of people affected by this inherited genetic disease, but perhaps also repairing those muscles.
The UW Medicine-led research focuses on delivering a series of protein packets inside a shuttle vector to replace the defective DMD gene within the muscles. The added genetic code will then start producing dystrophin, the protein lacking in patients with muscular dystrophy.
Geneticist Dr. Jeffery Chamberlain discusses the advance, shown to be effective in mouse-model tests, and his hopes to replicate that success in human clinical trials.
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