Arginase I mRNA Therapy – A Novel Approach to Rescue Arginase 1 Enzyme Deficiency


Authors: K.H. Asrani, L. Cheng, C.J. Cheng and R.R. Subramanian

Journal: RNA Biology

DOI: 10.1080/15476286.2018.1475178

Publication - Abstract

July 24, 2018

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.

Advanced Search

close
  • Publications
  • Application Notes
  • Posters
  • Workshops
  • Videos & Webinars
  • Articles
Search

Browse by Category

  • Application
    • Diagnostic and Imaging
    • Genetic Medicine
    • Hematology
    • Metabolic Disorders
    • Neuroscience
    • Oncology
    • Skeletal Disorders
    • Targeted Drug Delivery
    • Vaccines
    • Other Applications
    • Cell therapy
  • Formulation
    • Liposomes
    • Nucleic Acid Lipid Nanoparticles
    • Polymeric Nanoparticles
    • Other Formulations
  • Payload
    • DNA
    • microRNA
    • mRNA
    • siRNA
    • Small Molecule Drugs
    • Other Payloads


related content

Publication - Abstract

Humanized Biomimetic Nanovesicles for Neuron Targeting

A. Zinger, C. Cvetkovic, M. Sushnitha, T. Naoi, G. Baudo, M. Anderson, A. Shetty, N. Basu, J. Covello, E. Tasciotti, M. Amit, T. Xie, F. Taraballi, and R. Krencik

In an article written by Zinger et al. 2021 from the Center for Musculoskeletal Regeneration at the Houston Methodist Research Institute, the challenge of reproducible and scalable therapeutic drug delivery with high cell specificity to neural cells was investigated through analy...
Read More


Publication - Abstract

mRNA vaccines have become a promising platform for cancer immunotherapy. During vaccination, naked or vehicle loaded mRNA vaccines efficiently express tumor antigens in antigen-presenting cells (APCs), facilitate APC activation and innate/adaptive immune stimulation. mRNA cancer ...
Read More


Sign Up and Stay Informed
Sign up today to automatically receive new Precision NanoSystems application notes, conference posters, relevant science publications, and webinar invites.