Phenylalanine hydroxylase mRNA rescues the phenylketonuria phenotype in mice

Phenylketonuria (PKU) is an inborn error of metabolism caused by a deficiency in functional phenylalanine hydroxylase (PAH), resulting in accumulation of phenylalanine (Phe) in patients’ blood and organs. Affected patients encounter severe developmental delay, neurological deficits, and behavioral a...

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Detalles Bibliográficos
Autores principales: Cacicedo, Maximiliano L., Weinl-Tenbruck, Christine, Frank, Daniel, Limeres, Maria Jose, Wirsching, Sebastian, Hilbert, Katja, Pasha Famian, Mansure Abdollah, Horscroft, Nigel, Hennermann, Julia B., Zepp, Fred, Chevessier-Tünnesen, Frédéric, Gehring, Stephan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Bioengineering and Biotechnology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9585315/
https://www.ncbi.nlm.nih.gov/pubmed/36277393
http://dx.doi.org/10.3389/fbioe.2022.993298

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9585315/
https://www.ncbi.nlm.nih.gov/pubmed/36277393
http://dx.doi.org/10.3389/fbioe.2022.993298

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