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rAAV Gene Therapy for Myotonic Dystrophy
Utilizes rAAV Expression of Human Muscleblind ProteinsThis gene therapy employs recombinant adeno-associated viruses (rAAVs) that express human muscleblind proteins, treating myotonic dystrophy and other diseases caused by repeat microsatellite mutation expressions in coding and non-coding regions. Myotonic dystrophy is a dominantly inherited multi-systemic...
Published: 6/22/2026   |   Updated: 6/22/2026   |   Inventor(s): Charles Thornton, Rahul Kanadia, Maurice Swanson
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Category(s): Technology Classifications > Human Health Care > Therapeutics