Search Results - janice+chou

3 Results

Sort By:

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

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

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

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