Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency
Truong B, Allegri G, Liu XB, Burke KE, Zhu X, Cederbaum SD, Häberle J, Martini PGV, Lipshutz GS. Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency. Proc Natl Acad Sci U S A. 2019 Oct 15;116(42):21150-21159. doi: 10.1073/pnas.1906182116. Epub 2019 Sep 9. PMID: 31501335; PMCID: PMC6800360.