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Small Molecules That Facilitate Exon Skipping (UCLA Case No. 2014-258)

SUMMARYUCLA researchers in the Departments of Microbiology, Immunology, and Molecular Genetics, and Human Genetics have discovered a novel small molecule therapy that facilitates treatment of Duchene Muscular Dystrophy.BACKGROUNDDuchenne Muscular Dystrophy (DMD) is a genetic disorder caused by a mutation in the dystrophin gene. In DMD patients, muscles...

Published: 2/10/2024 | Inventor(s): Stanley Nelson, M. Carrie Miceli, Genevieve Kendall

Keywords(s): Musculoskeletal Disease, Therapeutics & Vaccines

Category(s): Therapeutics, Therapeutics > Musculoskeletal Disease