scholarly journals Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapy

Hepatology ◽  
2012 ◽  
Vol 56 (5) ◽  
pp. 1719-1729 ◽  
Author(s):  
Young Mok Lee ◽  
Hyun Sik Jun ◽  
Chi-Jiunn Pan ◽  
Su Ru Lin ◽  
Lane H. Wilson ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Hepatocellular Adenoma ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Metabolic Abnormalities ◽  
Type Ia

scholarly journals Downregulation of pathways implicated in liver inflammation and tumorigenesis of glycogen storage disease type Ia mice receiving gene therapy

2017 ◽  
Vol 26 (10) ◽  
pp. 1890-1899 ◽  
Author(s):  
Goo-Young Kim ◽  
Joon Hyun Kwon ◽  
Jun-Ho Cho ◽  
Lisa Zhang ◽  
Brian C. Mansfield ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Liver Inflammation ◽  
Type Ia

scholarly journals Fenofibrate rapidly decreases hepatic lipid and glycogen storage in neonatal mice with glycogen storage disease type Ia

2019 ◽  
Vol 29 (2) ◽  
pp. 286-294 ◽  
Author(s):  
Zollie A Yavarow ◽  
Hye-Ri Kang ◽  
Lauren R Waskowicz ◽  
Boon-Huat Bay ◽  
Sarah P Young ◽  
...  
Keyword(s):  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Hepatocellular Adenoma ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Pharmacological Therapy ◽  
Disease Type ◽  
Hepatic Glycogen ◽  
Alcoholic Fatty Liver ◽  
Type Ia

Abstract Glycogen storage disease type Ia (GSD Ia) is caused by autosomal mutations in glucose-6-phosphatase α catalytic subunit (G6PC) and can present with severe hypoglycemia, lactic acidosis and hypertriglyceridemia. In both children and adults with GSD Ia, there is over-accumulation of hepatic glycogen and triglycerides that can lead to steatohepatitis and a risk for hepatocellular adenoma or carcinoma. Here, we examined the effects of the commonly used peroxisomal proliferated activated receptor α agonist, fenofibrate, on liver and kidney autophagy and lipid metabolism in 5-day-old G6pc −/− mice serving as a model of neonatal GSD Ia. Five-day administration of fenofibrate decreased the elevated hepatic and renal triglyceride and hepatic glycogen levels found in control G6pc −/− mice. Fenofibrate also induced autophagy and promoted β-oxidation of fatty acids and stimulated gene expression of acyl-CoA dehydrogenases in the liver. These findings show that fenofibrate can rapidly decrease hepatic glycogen and triglyceride levels and renal triglyceride levels in neonatal G6pc −/− mice. Moreover, since fenofibrate is an FDA-approved drug that has an excellent safety profile, our findings suggest that fenofibrate could be a potential pharmacological therapy for GSD Ia in neonatal and pediatric patients as well as for adults. These findings may also apply to non-alcoholic fatty liver disease, which shares similar pathological and metabolic changes with GSD Ia.

scholarly journals Corrigendum to “Efficacy of Helper-dependent Adenovirus Vector-mediated Gene Therapy in Murine Glycogen Storage Disease Type Ia”

2007 ◽  
Vol 15 (11) ◽  
pp. 2051
Author(s):  
Dwight D Koeberl ◽  
Baodong Sun ◽  
Andrew Bird ◽  
Yuan-Tsong Chen ◽  
Kazuhiro Oka ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Adenovirus Vector ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Type Ia

scholarly journals Complete Normalization of Hepatic G6PC Deficiency in Murine Glycogen Storage Disease Type Ia Using Gene Therapy

2010 ◽  
Vol 18 (6) ◽  
pp. 1076-1084 ◽  
Author(s):  
Wai Han Yiu ◽  
Young Mok Lee ◽  
Wen-Tao Peng ◽  
Chi-Jiunn Pan ◽  
Paul A Mead ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Type Ia
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