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Improved Gene Therapy Vectors for the Treatment of Glycogen Storage Disease Type Ia (GSD-1a)

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, Barry Byrne

Keywords(s): Chou, GSD-1a, hepatic malignancy, hyperlipidemia, hypoglycemia, National Institute of Child Health and Human Development, NICHD

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