ArticleGene therapy2002
Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors.
Article in Gene therapy, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 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.
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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
24 citing papers in PubMed, 56 citations in OpenAlex.
- Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa).Journal of inherited metabolic disease · 2025Trial
- Integrative analysis of pathogenic variants in glucose-6-phosphatase based on an AlphaFold2 model.PNAS nexus · 2024Article
- Glycogen storage disease type I: Genetic etiology, clinical manifestations, and conventional and gene therapies.Pediatric discovery · 2023Article
- Dynamics of the Glycogen β-Particle Number in Rat Hepatocytes during Glucose Refeeding.International journal of molecular sciences · 2022Article
- Preclinical Research in Glycogen Storage Diseases: A Comprehensive Review of Current Animal Models.International journal of molecular sciences · 2020Review
- In search of proof-of-concept: gene therapy for glycogen storage disease type Ia.Journal of inherited metabolic disease · 2012Article
- Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia.Gene therapy · 2012Article
- Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia.Human gene therapy · 2012Article
- 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
- Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs.Human gene therapy · 2011Article
- Glycogen storage disease type Ia in canines: a model for human metabolic and genetic liver disease.Journal of biomedicine & biotechnology · 2011Review
- A detailed characterization of the adult mouse model of glycogen storage disease Ia.Laboratory investigation; a journal of technical methods and pathology · 2009Article
- Sexually dimorphic patterns of episomal rAAV genome persistence in the adult mouse liver and correlation with hepatocellular proliferation.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Gene therapy for lysosomal storage diseases (LSDs) in large animal models.ILAR journal · 2009Review
- Mutations in the glucose-6-phosphatase-alpha (G6PC) gene that cause type Ia glycogen storage disease.Human mutation · 2008Review
- Potential of AAV vectors in the treatment of metabolic disease.Gene therapy · 2008Review
- Gene therapy for type I glycogen storage diseases.Current gene therapy · 2007Review
- Glycogen storage disease types I and II: treatment updates.Journal of inherited metabolic disease · 2007Review
Corrections and comments
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Authors and funding
11 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Therapy in glycogen storage disease type Ia (GSD Ia), an inherited disorder of carbohydrate metabolism, relies on nutritional support that postpones but fails to prevent long-term complications of GSD Ia. In the canine model for GSD Ia, we evaluated the potential of intravenously delivered adeno-associated virus (AAV) vectors for gene therapy. In three affected canines, liver glycogen was reduced following hepatic expression of canine glucose-6-phosphatase (G6Pase). Two months after AAV vector administration, one affected dog had normalization of fasting glucose, cholesterol, triglycerides, and lactic acid. Concatamerized AAV vector DNA was confirmed by Southern blot analysis of liver DNA isolated from treated dogs, as head-to-tail, head-to-head, and tail-to-tail concatamers. Six weeks after vector administration, the level of vector DNA signal in each dog varied from one to five copies per cell, consistent with variation in the efficiency of transduction within the liver. AAV vector administration in the canine model for GSD Ia resulted in sustained G6Pase expression and improvement in liver histology and in biochemical parameters.
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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.