ArticleGene therapy2006
Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer.
Article in Gene therapy, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
34 citing papers in PubMed, 65 citations in OpenAlex.
- Gene therapy for liver diseases: methods, challenges and opportunities.Journal of nanobiotechnology · 2026Review
- Gene therapy for glycogen storage diseases.Journal of inherited metabolic disease · 2024Review
- Glycogen storage disease type I: Genetic etiology, clinical manifestations, and conventional and gene therapies.Pediatric discovery · 2023Article
- Gene therapy and genome editing for type I glycogen storage diseases.Frontiers in molecular medicine · 2023Review
- Preclinical Research in Glycogen Storage Diseases: A Comprehensive Review of Current Animal Models.International journal of molecular sciences · 2020Review
- Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia.Molecular therapy. Methods & clinical development · 2019Article
- Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia.Journal of inherited metabolic disease · 2018Article
- Nephron segment-specific gene expression using AAV vectors.Biochemical and biophysical research communications · 2018Article
- Recent development and gene therapy for glycogen storage disease type Ia.Liver research (Beijing, China) · 2017Article
- Renal rescue of dopamine D2 receptor function reverses renal injury and high blood pressure.JCI insight · 2016Article
- 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
- 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
- Targeted deletion of kidney glucose-6 phosphatase leads to nephropathy.Kidney international · 2014Article
- Development of hepatocellular adenomas and carcinomas in mice with liver-specific G6Pase-α deficiency.Disease models & mechanisms · 2014Article
- The threefold protrusions of adeno-associated virus type 8 are involved in cell surface targeting as well as postattachment processing.Journal of virology · 2012Article
- In search of proof-of-concept: gene therapy for glycogen storage disease type Ia.Journal of inherited metabolic disease · 2012Article
- Treatment of newborn G6pc(-/-) mice with bone marrow-derived myelomonocytes induces liver repair.Journal of hepatology · 2011Article
- Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Recombinant AAV-directed gene therapy for type I glycogen storage diseases.Expert opinion on biological therapy · 2011Review
- Perinatal gene transfer to the liver.Current pharmaceutical design · 2011Review
Corrections and comments
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Authors and funding
7 authors at 3 institutions in 1 country.
Funding
Abstract
Glycogen storage disease type Ia (GSD-Ia) is caused by a deficiency in glucose-6-phosphatase-alpha (G6Pase-alpha), a nine-transmembrane domain, endoplasmic reticulum-associated protein expressed primarily in the liver and kidney. Previously, we showed that infusion of an adeno-associated virus (AAV) serotype 2 vector carrying murine G6Pase-alpha (AAV2-G6Pase-alpha) into neonatal GSD-Ia mice failed to sustain their life beyond weaning. We now show that neonatal infusion of GSD-Ia mice with an AAV serotype 1-G6Pase-alpha (AAV1-G6Pase-alpha) or AAV serotype 8-G6Pase-alpha (AAV8-G6Pase-alpha) results in hepatic expression of the G6Pase-alpha transgene and markedly improves the survival of the mice. However, only AAV1-G6Pase-alpha can achieve significant renal transgene expression. A more effective strategy, in which a neonatal AAV1-G6Pase-alpha infusion is followed by a second infusion at age one week, provides sustained expression of a complete, functional, G6Pase-alpha system in both the liver and kidney and corrects the metabolic abnormalities in GSD-Ia mice for the 57 week length of the study. This effective use of gene therapy to correct metabolic imbalances and disease progression in GSD-Ia mice holds promise for the future of gene therapy in humans.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.