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...

Descripción completa

Detalles Bibliográficos
Autores principales: Daly, Owen, Mahiny, Azita Josefine, Majeski, Sara, McClintock, Kevin, Reichert, Julia, Boros, Gábor, Szabó, Gábor Tamás, Reinholz, Jonas, Schreiner, Petra, Reid, Steve, Lam, Kieu, Lepper, Marlen, Adler, Melanie, Meffen, Tracy, Heyes, James, Karikó, Katalin, Lutwyche, Pete, Vlatkovic, Irena
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10296609/
https://www.ncbi.nlm.nih.gov/pubmed/37371829
http://dx.doi.org/10.3390/biomedicines11061735

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10296609/
https://www.ncbi.nlm.nih.gov/pubmed/37371829
http://dx.doi.org/10.3390/biomedicines11061735

Ejemplares similares