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2020-781 GENE THERAPY VECTORS FOR TREATING LIMB GIRDLE MUSCULAR DYSTROPHY

Researchers in the Departments of Neurology at UCLA and the University of Washington have developed a novel AAV gene therapy that treats limb-girdle muscular dystrophy type R1/2A (LGMD2A), an as of yet unmet target.BACKGROUND:Muscular dystrophies are a group of muscle diseases caused by mutations in a persons’ genes. Mutations within the calpain3...

Published: 7/19/2023 | Inventor(s): Melissa Spencer, Irina Kramerova, Steve Hauschka, Jeffrey Chamberlain

Keywords(s): Gene Therapy, Muscular Dystrophy

Category(s): Therapeutics > Gene Therapy And Editing