Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing

Phenylketonuria (PKU) is a metabolic disorder whereby phenylalanine metabolism is deficient due to allelic variations in the gene for phenylalanine hydroxylase (PAH). There is no cure for PKU other than orthotopic liver transplantation, and the standard of care for patients is limited to dietary res...

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Detalles Bibliográficos
Autores principales: Kaiser, Robert A., Carlson, Daniel F., Allen, Kari L., Webster, Dennis A., VanLith, Caitlin J., Nicolas, Clara T., Hillin, Lori G., Yu, Yue, Kaiser, Catherine W., Wahoff, William R., Hickey, Raymond D., Watson, Adrienne L., Winn, Shelley R., Thöny, Beat, Kern, Douglas R., Harding, Cary O., Lillegard, Joseph B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2021
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7833140/
https://www.ncbi.nlm.nih.gov/pubmed/33493163
http://dx.doi.org/10.1371/journal.pone.0245831

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