Use of an adeno‐associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model

Phenylketonuria (PKU) is the most common inborn error of metabolism of the liver, and results from mutations of both alleles of the phenylalanine hydroxylase gene (PAH). As such, it is a suitable target for gene therapy via gene delivery with a recombinant adeno‐associated virus (AAV) vector. Here w...

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Detalles Bibliográficos
Autores principales: Kaiser, Robert A., Weber, Nicholas D., Trigueros‐Motos, Laia, Allen, Kari L., Martinez, Michael, Cao, William, VanLith, Caitlin J., Hillin, Lori G., Douar, Anne, González‐Aseguinolaza, Gloria, Aldabe, Rafael, Lillegard, Joseph B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9291745/
https://www.ncbi.nlm.nih.gov/pubmed/33896013
http://dx.doi.org/10.1002/jimd.12392

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9291745/
https://www.ncbi.nlm.nih.gov/pubmed/33896013
http://dx.doi.org/10.1002/jimd.12392

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