ArticleRNA biology2018
Arginase I mRNA therapy - a novel approach to rescue arginase 1 enzyme deficiency.
Article in RNA biology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 56 citations in OpenAlex.
- Retrospective analysis of arginase 1 deficiency progression in adults over 5 years at a single metabolic centre.JIMD reports · 2024Article
- The therapeutic landscape of citrin deficiency.Journal of inherited metabolic disease · 2024Review
- Unleashing the potential of mRNA therapeutics for inherited neurological diseases.Brain : a journal of neurology · 2024Review
- Nanoparticle-Mediated Mucosal Vaccination: Harnessing Nucleic Acids for Immune Enhancement.Current microbiology · 2024Review
- Hyperargininemia: A Rare Diagnosis in Adulthood.European journal of case reports in internal medicine · 2024Article
- Treatment and management for children with urea cycle disorder in chronic stage.Zhejiang da xue xue bao. Yi xue ban = Journal of Zhejiang University. Medical sciences · 2023Article
- What Can RNA-Based Therapy Do for Monogenic Diseases?Pharmaceutics · 2023Review
- Human arginase 1, a Jack of all trades?3 Biotech · 2022Review
- Natural history of arginase 1 deficiency and the unmet needs of patients: A systematic review of case reports.JIMD reports · 2022Article
- mRNA vaccines: A novel weapon to control infectious diseases.Frontiers in microbiology · 2022Review
- The Pivotal Role of Chemical Modifications in mRNA Therapeutics.Frontiers in cell and developmental biology · 2022Review
- Non-Immunotherapy Application of LNP-mRNA: Maximizing Efficacy and Safety.Biomedicines · 2021Review
- Novel Homozygous Missense Mutation in theFrontiers in neurology · 2020Article
- The promise of mRNA vaccines: a biotech and industrial perspective.NPJ vaccines · 2020Review
- Messenger RNA therapy as an option for treating metabolic disorders.Proceedings of the National Academy of Sciences of the United States of America · 2019Article
- Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Arginase I (ARG1) deficiency is an autosomal recessive urea cycle disorder, caused by deficiency of the enzyme Arginase I, resulting in accumulation of arginine in blood. Current Standard of Care (SOC) for ARG1 deficiency in patients or those having detrimental mutations of ARG1 gene is diet control. Despite diet and drug therapy with nitrogen scavengers, ~25% of patients suffer from severe mental deficits and loss of ambulation. 75% of patients whose symptoms can be managed through diet therapy continue to suffer neuro-cognitive deficits. In our research, we demonstrate in vitro and in vivo that administration of ARG1 mRNA increased ARG1 protein expression and specific activity in relevant cell types, including ARG1-deficient patient cell lines, as well as in wild type mice for up to 4 days. These studies demonstrate that ARG1 mRNA treatment led to increased functional protein expression of ARG1 and subsequently an increase in urea. Hence, ARG1 mRNA therapy could be a potential treatment option to develop for patients.
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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.