G6PC mRNA Therapy Positively Regulates Fasting Blood Glucose and Decreases Liver Abnormalities in a Mouse Model of Glycogen Storage Disease 1a

Author:

Roseman Daniel S.,Khan Tayeba,Rajas Fabienne,Jun Lucy S.,Asrani Kirtika H.,Isaacs Cleo,Farelli Jeremiah D.,Subramanian Romesh R.

Funder

Alexion Pharmaceuticals

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference26 articles.

1. Isolation of the gene for murine glucose-6-phosphatase, the enzyme deficient in glycogen storage disease type 1A;Shelly;J. Biol. Chem.,1993

2. Glucose-6-phosphatase deficiency;Froissart;Orphanet J. Rare Dis.,2011

3. Lessons from new mouse models of glycogen storage disease type 1a in relation to the time course and organ specificity of the disease;Rajas;J. Inherit. Metab. Dis.,2015

4. Glycogen storage disease type I and G6Pase-β deficiency: etiology and therapy;Chou;Nat. Rev. Endocrinol.,2010

5. Enzymatic characterization of four new mutations in the glucose-6 phosphatase (G6PC) gene which cause glycogen storage disease type 1a;Bruni;Ann. Hum. Genet.,1999

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