Search Results - gerald+lipshutz

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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

2019-600 TREATMENT FOR RESTORING UREAGENESIS IN CARBAMOYL PHOSPHATE SYNTHETASE 1 DEFICIENCY

Treatment for Restoring Ureagenesis in Carbamoyl Phosphate Synthetase 1 DeficiencyTech ID: 30356 / UC Case 2019-600-0SUMMARYUCLA researchers in the Department of Surgery have developed a gene therapy to treat carbamoyl phosphate synthetase 1 deficiency.BACKGROUNDCarbamoyl phosphate synthetase 1 (CPS1) deficiency is an inherited autosomal recessive urea...

Published: 9/5/2023 | Inventor(s): Gerald Lipshutz

Keywords(s): Cns, Gene Editing Systems, Gene Therapy, Genetic Disease, Therapeutics & Vaccines

Category(s): Therapeutics, Therapeutics > CNS and Neurology, Therapeutics > Gene Therapy And Editing

2019-550: A Method to Prevent the Myelin Abnormalities Associated with Arginase Deficiency

SUMMARY UCLA researchers in the Department of Surgery have developed a gene therapy to prevent dysmyelination (and other CNS abnormalities) as a result of arginase deficiency. BACKGROUND Arginase deficiency (ARG1 deficiency, argininemia) is an inherited autosomal recessive disorder caused by deficient/defective enzyme arginase 1 in the final...

Published: 9/5/2023 | Inventor(s): Gerald Lipshutz

Keywords(s): Cns, Gene Editing Systems, Gene Therapy, Genetic Disease, Therapeutics & Vaccines

Category(s): Therapeutics, Therapeutics > CNS and Neurology, Therapeutics > Gene Therapy And Editing