ArticleMolecular therapy : the journal of the American Society of Gene Therapy2011
Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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23 citing papers in PubMed, 36 citations in OpenAlex.
- Novel promoters drive therapeutic transgene expression and evade transgene-specific immune responses in a mouse model of Pompe disease.Molecular genetics and metabolism · 2026Article
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- Gene therapy for glycogen storage diseases.Journal of inherited metabolic disease · 2024Review
- Genome editing usingMolecular therapy. Methods & clinical development · 2023Article
- Glycogen storage disease type I: Genetic etiology, clinical manifestations, and conventional and gene therapies.Pediatric discovery · 2023Article
- Glycogen storage disease type 1a in the Ohio Amish.JIMD reports · 2022Article
- [Advances on the management of renal lesion in glycogen storage disease type I].Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology · 2021Article
- Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease.Molecular therapy. Methods & clinical development · 2019Article
- Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia.Molecular therapy. Methods & clinical development · 2019Article
- Long-term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapy.Journal of inherited metabolic disease · 2018Article
- In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Large animal models and new therapies for glycogen storage disease.Journal of inherited metabolic disease · 2015Review
- Lessons from new mouse models of glycogen storage disease type 1a in relation to the time course and organ specificity of the disease.Journal of inherited metabolic disease · 2015Review
- Adeno-associated virus-mediated cancer gene therapy: current status.Cancer letters · 2015Review
- Preclinical Development of New Therapy for Glycogen Storage Diseases.Current gene therapy · 2015Review
- Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade.Human gene therapy · 2015Article
- Targeted deletion of kidney glucose-6 phosphatase leads to nephropathy.Kidney international · 2014Article
- Kidney-specific expression of GFP by in-utero delivery of pseudotyped adeno-associated virus 9.Molecular therapy. Methods & clinical development · 2014Article
- The upstream enhancer elements of the G6PC promoter are critical for optimal G6PC expression in murine glycogen storage disease type Ia.Molecular genetics and metabolism · 2013Article
- Multiple recombinant adeno-associated viral vector serotypes display persistent in vivo gene expression in vector-transduced rat stifle joints.Human gene therapy methods · 2013Article
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9 authors at 3 institutions in 1 country.
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No grant is acknowledged in the PubMed record.
Abstract
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 encoding human G6Pase was pseudotyped with four serotypes, AAV2, AAV7, AAV8, and AAV9, and we evaluated efficacy in 12-day-old G6pase (-/-) mice. Hypoglycemia during fasting (plasma glucose <100 mg/dl) was prevented for >6 months by the dsAAV2/7, dsAAV2/8, and dsAAV2/9 vectors. Prolonged fasting for 8 hours revealed normalization of blood glucose following dsAAV2/9 vector administration at the higher dose. The glycogen content of kidney was reduced by >65% with both the dsAAV2/7 and dsAAV2/9 vectors, and renal glycogen content was stably reduced between 7 and 12 months of age for the dsAAV2/9 vector-treated mice. Every vector-treated group had significantly reduced glycogen content in the liver, in comparison with untreated G6pase (-/-) mice. G6Pase was expressed in many renal epithelial cells of with the dsAAV2/9 vector for up to 12 months. Albuminuria and renal fibrosis were reduced by the dsAAV2/9 vector. Hepatorenal correction in G6pase (-/-) mice demonstrates the potential of AAV vectors for the correction of inherited diseases of metabolism.
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