Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing

Phenylketonuria (PKU), an autosomal recessive disorder caused by pathogenic variants in the phenylalanine hydroxylase (PAH) gene, results in the accumulation of blood phenylalanine (Phe) to neurotoxic levels. Current dietary and medical treatments are chronic and reduce, rather than normalize, blood...

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Detalles Bibliográficos
Autores principales: Brooks, Dominique L., Carrasco, Manuel J., Qu, Ping, Peranteau, William H., Ahrens-Nicklas, Rebecca C., Musunuru, Kiran, Alameh, Mohamad-Gabriel, Wang, Xiao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10257655/
https://www.ncbi.nlm.nih.gov/pubmed/37301931
http://dx.doi.org/10.1038/s41467-023-39246-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10257655/
https://www.ncbi.nlm.nih.gov/pubmed/37301931
http://dx.doi.org/10.1038/s41467-023-39246-2

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