Search Results - gene+delivery

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Methods and Compositions for Modification of the HPRT Locus (UCLA Case No. 2013-570)

UCLA researchers have developed a gene editing strategy and methodology based on nuclease-based insertion. 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 implement this technology....

Published: 4/12/2024 | Inventor(s): Noriyuki Kasahara

Keywords(s): Gene delivery, Gene Editing Systems, Gene Therapy

Category(s): Therapeutics > Gene Therapy And Editing

Gene Augmentation Therapy for Usher Syndrome 1B (UCLA Case No. 2023-138)

UCLA researchers in the Department of Ophthalmology have developed a novel approach to cure Usher Syndrome 1B. BACKGROUND: Usher syndrome is defined as a condition whose major symptoms are deafness or hearing loss, and an eye disease called retinitis pigmentosa (RP), which affects vision. The ear-related effects of Usher’s disease are caused...

Published: 9/15/2023 | Inventor(s): David Williams

Keywords(s): Gene delivery, Genetic Disease, Hearing loss, Usher syndrome, Vision loss

Category(s): Therapeutics > Gene Therapy And Editing