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Recombinant Virus Vectors for the Treatment of Glycogen Storage Disease type Ib (GSD-Ib)

Abstract: Glycogen storage disease type Ib (GSD-Ib) is an autosomal recessive disorder caused by deficiencies in glucose-6-phosphate transporter (G6PT), a ubiquitously expressed endoplasmic reticulum (ER) protein that translocates G6P from the cytoplasm into the ER lumen. Inside the ER, G6P is hydrolyzed to glucose and phosphate by either the liver/kidney/intestine-restricted...

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

Keywords(s): autosomal recessive disorder, Chou, G6PT, GENE THERAPY, glucose homeostasis, Glycogen storage disease type Ib, GSD-Ib, hepatocellular adenoma, metabolic and immune disorders, myeloid dysfunction, NEUTROPENIA, NICHD

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