Search Results - stanley+nelson

2 Results

Sort By:

Small Molecules to Facilitate Therapeutic Exon Skipping (UCLA Case No. 2009-381)

Summary Investigators at UCLA have discovered a series of small molecules to facilitate therapeutic exon skipping. The use of these identified molecules may enhance the effectiveness of antisense oligonucleotide agents currently in clinical development. Background A number of antisense oligonucleotide agents are currently in clinical trials for a wide...

Published: 7/17/2025 | Inventor(s): Stanley Nelson

Keywords(s):

Category(s): Platforms > Drug Delivery > Nucleic Acid Platform Systems

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: 7/17/2025 | Inventor(s): Stanley Nelson, M Miceli, Genevieve Kendall

Keywords(s): Musculoskeletal Disease, Therapeutics & Vaccines

Category(s): Therapeutics, Therapeutics > Musculoskeletal Disease