ArticleMolecular therapy : the journal of the American Society of Gene Therapy2009
Short-term correction of arginase deficiency in a neonatal murine model with a helper-dependent adenoviral vector.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
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Who cites it
25 citing papers in PubMed, 30 citations in OpenAlex.
- Gene therapy for urea cycle defects: An update from historical perspectives to future prospects.Journal of inherited metabolic disease · 2024Review
- Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency.Molecular therapy. Nucleic acids · 2022Article
- Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency.Proceedings of the National Academy of Sciences of the United States of America · 2019Article
- Hepatic arginase deficiency fosters dysmyelination during postnatal CNS development.JCI insight · 2019Article
- Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice.Molecular genetics and metabolism · 2018Article
- Arginase I mRNA therapy - a novel approach to rescue arginase 1 enzyme deficiency.RNA biology · 2018Article
- Proof-of-Concept Gene Editing for the Murine Model of Inducible Arginase-1 Deficiency.Scientific reports · 2017Article
- Liver-specific knockout of arginase-1 leads to a profound phenotype similar to inducible whole body arginase-1 deficiency.Molecular genetics and metabolism reports · 2016Article
- Restoring Ureagenesis in Hepatocytes by CRISPR/Cas9-mediated Genomic Addition to Arginase-deficient Induced Pluripotent Stem Cells.Molecular therapy. Nucleic acids · 2016Article
- Rescue of the Functional Alterations of Motor Cortical Circuits in Arginase Deficiency by Neonatal Gene Therapy.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2016Article
- Arginase-1 deficiency.Journal of molecular medicine (Berlin, Germany) · 2015Review
- Development of operational immunologic tolerance with neonatal gene transfer in nonhuman primates: preliminary studies.Gene therapy · 2015Article
- Minimal ureagenesis is necessary for survival in the murine model of hyperargininemia treated by AAV-based gene therapy.Gene therapy · 2015Article
- Article
- Myocyte-mediated arginase expression controls hyperargininemia but not hyperammonemia in arginase-deficient mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Combinatorial treatment with oncolytic adenovirus and helper-dependent adenovirus augments adenoviral cancer gene therapy.Molecular therapy oncolytics · 2014Article
- Innate functions of immunoglobulin M lessen liver gene transfer with helper-dependent adenovirus.PloS one · 2014Article
- Lethal phenotype in conditional late-onset arginase 1 deficiency in the mouse.Molecular genetics and metabolism · 2013Article
- Inducible arginase 1 deficiency in mice leads to hyperargininemia and altered amino acid metabolism.PloS one · 2013Article
- Long-term survival of the juvenile lethal arginase-deficient mouse with AAV gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
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Authors and funding
11 authors at 3 institutions in 2 countries.
Funding
Abstract
Neonatal gene therapy has the potential to ameliorate abnormalities before disease onset. Our gene knockout of arginase I (AI) deficiency is characterized by increasing hyperammonemia, neurological deterioration, and early death. We constructed a helper-dependent adenoviral vector (HDV) carrying AI and examined for correction of this defect. Neonates were administered 5 x 10(9) viral particles/g and analyzed for survival, arginase activity, and ammonia and amino acids levels. The life expectancy of arg(-/-) mice increased to 27 days while controls died at 14 days with hyperammonemia and in extremis. Death correlated with a decrease in viral DNA/RNA per cell as liver mass increased. Arginase assays demonstrated that vector-injected hepatocytes had ~20% activity of heterozygotes at 2 weeks of age. Hepatic arginine and ornithine in treated mice were similar to those of saline-injected heterozygotes at 2 weeks, whereas ammonia was normal. By 26 days, arginase activity in the treated arg(-/-) livers declined to <10%, and arginine and ornithine increased. Ammonia levels began increasing by day 25, suggesting the cause of death to be similar to that of uninjected arg(-/-) mice, albeit at a later time. These studies demonstrate that the AI deficient newborn mouse can be temporarily corrected and rescued using a HDV.
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