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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