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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

2017-701 Nanoparticle-Enabled Delivery of Large Plasmid DNA for Gene Therapy of Duchenne Muscular Dystrophy and Cancer

Polyrotaxane Nanoparticles for Delivery of Large Plasmid DNA in Duchenne Muscular DystrophySUMMARYUCLA researchers have designed, synthesized, and validated a polyrotaxane nanocarrier for targeted delivery of large plasmids for gene therapy applications for treatment of Duchenne muscular dystrophy and cancer.BACKGROUNDRapid development of genome/epigenome...

Published: 7/19/2023 | Inventor(s): Melissa Spencer, April Pyle, Huan Meng, Courtney Young, Xiangsheng Liu

Keywords(s): Drug Delivery, Gene Editing Systems, Gene Therapy, Genetic Disease, Life Science Research Tools, Medical Devices and Materials, Musculoskeletal Disease, Nanoparticles, Oncology, PEG, Therapeutics & Vaccines

Category(s): Life Science Research Tools, Medical Devices, Platforms > Drug Delivery, Therapeutics, Therapeutics > Gene Therapy And Editing, Therapeutics > Musculoskeletal Disease, Platforms > Drug Delivery > Nanoparticles, Therapeutics > Oncology, Platforms > Drug Delivery > PEG