Search Results - therapeutics+%3e+genetic+diseases

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Augmentations to Lentiviral Vectors to Increase Expression (UCLA Case No. 2019-616)

UCLA researchers in the Department of Microbiology, Immunology and Molecular Genetics have developed a novel method to produce short lentiviral vectors with tissue-specific expression, with a primary focus on lentiviral vectors for treating sickle cell disease and other disorders of hemoglobin. BACKGROUND: Sickle cell disease (SCD) is one of the...

Published: 8/14/2024 | Inventor(s): Donald Kohn, Roger Hollis, Richard Morgan

Keywords(s):

Category(s): Therapeutics > Gene Therapy And Editing, Life Science Research Tools, Therapeutics > Genetic Diseases

Pharmacologic Intervention to Reduce the Risk of Episodic Weakness and Enhance Recovery from On-Going Episode of Weakness in Periodic Paralysis (UCLA Case No. 2022-274)

Researchers in the Department of Physiology in the School of Medicine have identified a novel method for the treatment of periodic paralysis. This approach focuses on a type of potassium channel that the team has newly discovered which can be modulated to relieve or prevent episodes of periodic paralysis with an FDA-approved drug. BACKGROUND: Periodic...

Published: 2/23/2024 | Inventor(s): Stephen Cannon

Keywords(s): Drug repurposing, Muscle weakness, Paralysis, Periodic paralysis

Category(s): Medical Devices, Therapeutics > Musculoskeletal Disease, Therapeutics > Genetic Diseases

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