Search Results - melissa+spencer

4 Results

Sort By:

TRP: A Mouse Model of Limb Girdle Muscular Dystrophy Type 2A (R1/D4) (UCLA Case No. 2021-171)

UCLA researchers in the Department of Neurology have developed a novel mouse model for limb girdle muscular dystrophy, a disorder that causes muscle weakness in the limbs and hip area. BACKGROUND: Limb-Girdle Muscular Dystrophy Type 2A (LGMDR1) is a genetic neuromuscular disorder characterized by progressive muscle weakness, primarily affecting muscles...

Published: 7/30/2025 | Inventor(s): Melissa Spencer

Keywords(s):

Category(s): Life Science Research Tools > Animal Models

Novel AAV Serotypes Derived From a Library Screen (UCLA Case No. 2020-789)

UCLA researchers in the Department of Neurology have produced novel AAV serotypes for a wide range of tissue and cell specific gene delivery. BACKGROUND: Gene therapies are the forefront of therapeutic advancements for debilitating diseases. However, delivery and insertion of the therapeutic transgene are major hurdles that must be solved to effectively...

Published: 7/17/2025 | Inventor(s): Melissa Spencer

Keywords(s):

Category(s): Therapeutics > Gene Therapy And Editing

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: 7/17/2025 | 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: 7/17/2025 | 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