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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