ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014
Myocyte-mediated arginase expression controls hyperargininemia but not hyperammonemia in arginase-deficient mice.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2014. 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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Who cites it
23 citing papers in PubMed.
- Adeno-Associated virus-based approaches for mitochondrial diseases: advances and challenges.Molecular psychiatry · 2026Review
- Extracellular Vesicles Delivered a Functional ARG1 Enzyme and Restored Its Activity in a Mouse Model of ARG1-D Resulting in Improved Lifespan.International journal of molecular sciences · 2026Article
- Murine toxicology assessment of avgn7.2, a novel gene therapeutic for inclusion body myositis and other muscle wasting diseases.Gene therapy · 2026Article
- Muscle-specific gene editing improves molecular and phenotypic defects in a mouse model of myotonic dystrophy type 1.Clinical and translational medicine · 2025Article
- Efficacy and muscle safety assessment of fukutin-related protein gene therapy.Molecular therapy. Methods & clinical development · 2023Article
- Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency.Molecular therapy. Nucleic acids · 2022Article
- Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities.Molecular therapy. Methods & clinical development · 2022Article
- A universal strategy for AAV delivery of base editors to correct genetic point mutations in neonatal PKU mice.Molecular therapy. Methods & clinical development · 2022Article
- Micro-dystrophin Gene Therapy Partially Enhances Exercise Capacity in Older AdultMolecular therapy. Methods & clinical development · 2020Article
- 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
- Proof-of-Concept Gene Editing for the Murine Model of Inducible Arginase-1 Deficiency.Scientific reports · 2017Article
- Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.Nature communications · 2017Article
- Functional Human and Murine Tissue-Engineered Liver Is Generated from Adult Stem/Progenitor Cells.Stem cells translational medicine · 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
- Progress and prospects of gene therapy clinical trials for the muscular dystrophies.Human molecular genetics · 2016Review
- Human recombinant arginase enzyme reduces plasma arginine in mouse models of arginase deficiency.Human molecular genetics · 2015Article
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Abstract
Human arginase deficiency is characterized by hyperargininemia and infrequent episodes of hyperammonemia that cause neurological impairment and growth retardation. We previously developed a neonatal mouse adeno-associated viral vector (AAV) rh10-mediated therapeutic approach with arginase expressed by a chicken β-actin promoter that controlled plasma ammonia and arginine, but hepatic arginase declined rapidly. This study tested a codon-optimized arginase cDNA and compared the chicken β-actin promoter to liver- and muscle-specific promoters. ARG1(-/-) mice treated with AAVrh10 carrying the liver-specific promoter also exhibited long-term survival and declining hepatic arginase accompanied by the loss of AAV episomes during subsequent liver growth. Although arginase expression in striated muscle was not expected to counteract hyperammonemia, due to muscle's lack of other urea cycle enzymes, we hypothesized that the postmitotic phenotype in muscle would allow vector genomes to persist, and hence contribute to decreased plasma arginine. As anticipated, ARG1(-/-) neonatal mice treated with AAVrh10 carrying a modified creatine kinase-based muscle-specific promoter did not survive longer than controls; however, their plasma arginine levels remained normal when animals were hyperammonemic. These data imply that plasma arginine can be controlled in arginase deficiency by muscle-specific expression, thus suggesting an alternative approach to utilizing the liver for treating hyperargininemia.
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