ASL mRNA-LNP Therapeutic for the Treatment of Argininosuccinic Aciduria Enables Survival Benefit in a Mouse Model

ASL mRNA-LNP Therapeutic for the Treatment of Argininosuccinic Aciduria Enables Survival Benefit in a Mouse Model

Argininosuccinic aciduria (ASA) is a metabolic disorder caused by a deficiency in argininosuccinate lyase (ASL), which cleaves argininosuccinic acid to arginine and fumarate in the urea cycle. ASL deficiency (ASLD) leads to hepatocyte dysfunction, hyperammonemia, encephalopathy, and respiratory alka...

Full description

Bibliographic Details
Main Authors: Owen Daly, Azita Josefine Mahiny, Sara Majeski, Kevin McClintock, Julia Reichert, Gábor Boros, Gábor Tamás Szabó, Jonas Reinholz, Petra Schreiner, Steve Reid, Kieu Lam, Marlen Lepper, Melanie Adler, Tracy Meffen, James Heyes, Katalin Karikó, Pete Lutwyche, Irena Vlatkovic
Format: Article
Language:English
Published: MDPI AG 2023-06-01
Series:Biomedicines
Subjects:
lipid nanoparticle-mRNA (LNP-mRNA)
mRNA optimization
mRNA therapeutic
rare disease
argininosuccinic aciduria (ASA)
argininosuccinate lyase deficiency (ASLD)
Online Access:https://www.mdpi.com/2227-9059/11/6/1735

Internet

https://www.mdpi.com/2227-9059/11/6/1735

Similar Items