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Gene Therapy Vector for the Treatment of Glycogen Storage Disease Type Ia (GSD-Ia)

Abstract: GSD-Ia is an inherited disorder of metabolism associated with life-threatening hypoglycemia, hepatic malignancy, and renal failure caused by the deficiency of glucose-6-phosphatase-alpha (G6Pase-alpha or G6PC). Current therapy, which primarily consists of dietary modification, fails to prevent long-term complications in many patients, including...

Published: 4/8/2024 | Inventor(s): Janice Chou

Keywords(s): GSD-Ia, hepatic malignancy, hypoglycemia, renal failure

Category(s): Collaboration Sought > Licensing, TherapeuticArea > Nephrology, Collaboration Sought > Collaboration, Application > Therapeutics