A schematic illustration of a multi-shuttle system to deliver full dystrophin protein to muscle cells body-wide as a genetic therapy for muscular dystrophy. Credit: Hichem Tasfaout A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles. The UW Medicine-led research focuses on delivering a series of protein packets inside shuttle vectors to replace the defective DMD gene within the muscles. The added genetic code will then start producing dystrophin, the protein lacking in patients Read More
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