Search Results - adeno-associated+viruses+(aav)

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CHST6 Mini Gene - CHST6 Expression Construct With Native Promoter (UCLA Case No. 2023-301)

UCLA researchers from the Department of Human Genetics and Department of Ophthalmology have developed a mini gene encoding a protein which is mutated in macular corneal dystrophy, leading to a potential gene therapy for this disorder. BACKGROUND: Macular corneal dystrophy (MCD) is a rare genetic disease caused by mutations in the CHST6 gene, which...

Published: 11/20/2024 | Inventor(s): Wenlin Zhang, Anthony Aldave

Keywords(s): AAV, Adeno-associated viruses (AAV), Gene Editing Systems, Gene Therapy, genetics, keratinocytes, macular corneal dystrophy, mini gene, mutation, native promoter, Ophthalmology

Category(s): Therapeutics > Ophthalmology, Therapeutics > Gene Therapy And Editing

AAV-Based Treatment of Guanidinoacetate Methyltransferase (GAMT) Deficiency (UCLA Case No. 2021-012)

SUMMARY: UCLA researchers in the Departments Molecular & Medical Pharmacology and Surgery in the David Geffen School of Medicine have developed a novel and tissue-specific gene therapy treatment for Guanidinoacetate methyltransferase (GAMT) deficiency. BACKGROUND: Guanidinoacetate Methyltransferase (GAMT) deficiency is caused by a mutation in...

Published: 8/10/2023 | Inventor(s): Gerald Lipshutz

Keywords(s): Adeno-associated viruses (AAV), Creatine, GAMT deficiency, Gene Therapy, Metabolic Diseases

Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Genetic Diseases, Therapeutics > Metabolism And Endocrinology