Hepatorenal Correction in Murine Glycogen Storage Disease Type I With a Double-stranded Adeno-associated Virus Vector

Glycogen storage disease type Ia (GSD-Ia) is caused by the deficiency of glucose-6-phosphatase (G6Pase). Long-term complications of GSD-Ia include life-threatening hypoglycemia and proteinuria progressing to renal failure. A double-stranded (ds) adeno-associated virus serotype 2 (AAV2) vector encodi...

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Veröffentlicht in:	Molecular therapy 2011-11, Vol.19 (11), p.1961-1970
Hauptverfasser:	Luo, Xiaoyan, Hall, Gentzon, Li, Songtao, Bird, Andrew, Lavin, Peter J, Winn, Michelle P, Kemper, Alex R, Brown, Talmage T, Koeberl, Dwight D
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno-associated virus Age Animals Cholesterol Dependovirus - genetics Disease Models, Animal Drug dosages Female Gene Expression Regulation Gene therapy Genetic Therapy Genetic Vectors - administration & dosage Genetic Vectors - genetics Genetics Glucose Glucose-6-Phosphatase - genetics Glycogen Storage Disease Type I - genetics Glycogen Storage Disease Type I - mortality Glycogen Storage Disease Type I - therapy Humans Hypoglycemia Hypoglycemia - genetics Hypoglycemia - therapy Kaplan-Meier Estimate Kidney - metabolism Kidney diseases Liver - metabolism Liver cancer Male Metabolism Mice Mice, Knockout Original Pediatrics Phosphatase Plasma Survival analysis Tumors Vectors (Biology)
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