Lipid nanoparticle delivers phenylalanine ammonia lyase mRNA to the liver leading to catabolism and clearance of phenylalanine in a phenylketonuria mouse model

Phenylketonuria (PKU) is a genetic disorder affecting around 1 in 12,000 live births (1), caused by a mutation in the phenylalanine hydroxylase (PAH) gene in the liver which facilitates the catabolism of phenylalanine (Phe). Without a functional copy of PAH, levels of Phe in the blood and tissues ri...

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Detalles Bibliográficos
Autores principales: Diaz-Trelles, Ramon, Lee, Sharon, Kuakini, Kristen, Park, Jenny, Dukanovic, Adrian, Gonzalez, Jose A., Dam, Thanhchau, Kim, Jae Heon, Vega, Jerel Boyd, Sablad, Marciano, Karmali, Priya P., Tachikawa, Kiyoshi, Chivukula, Padmanabh
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Short Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9118528/
https://www.ncbi.nlm.nih.gov/pubmed/35600090
http://dx.doi.org/10.1016/j.ymgmr.2022.100882

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