image: This is a microscopic image of the liver showing arginase mRNA delivered by lipid nanoparticle as small brown areas within hepatocytes. view more
Credit: Image courtesy of Kristine E. Burke
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
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Journal
Proceedings of the National Academy of Sciences