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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
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Inventor(s):
Melissa Spencer
,
Irina Kramerova
,
Steve Hauschka
,
Jeffrey Chamberlain
Keywords(s):
Gene Therapy
,
Muscular Dystrophy
Category(s):
Therapeutics > Gene Therapy And Editing
Exercise In A Pill: Compounds That Reproduce The Effects Of Exercise On Muscle Metabolism And Growth
SummaryUCLA researchers in the Department of Neurology have identified and synthesized small molecule analogs that activate skeletal muscle growth, mediated by calcium calmodulin kinase II signaling.BackgroundSkeletal muscle has a remarkable ability to alter its metabolic and contractile phenotype in response to changes in functional demands. Increased...
Published: 7/19/2023
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Inventor(s):
Varghese John
,
Irina Kramerova
Keywords(s):
Musculoskeletal Disease
,
Therapeutics & Vaccines
Category(s):
Therapeutics
,
Therapeutics > Musculoskeletal Disease