Genetic deficiency of the liver enzyme arginase interrupts the urea cycle and causes a range of symptoms in humans, and a study finds that in mice with a genetic arginase deficiency, administration of liver-targeted nanoparticles that carried a messenger RNA for the arginase gene resulted in restored urea cycle function, ammonium metabolism, and prolonged lifespan.
Article #19-06182: "Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency," by Brian Truong et al.
MEDIA CONTACT: Gerald S. Lipshutz, University of California, Los Angeles, CA; e-mail: glipshutz@mednet.ucla.edu
###
Proceedings of the National Academy of Sciences