AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

Phenylketonuria (PKU) due to recessively inherited phenylalanine hydroxylase (PAH) deficiency results in hyperphenylalaninemia, which is toxic to the central nervous system. Restriction of dietary phenylalanine intake remains the standard of PKU care and prevents the major neurologic manifestations...

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Detalles Bibliográficos
Autores principales: Richards, Daelyn Y., Winn, Shelley R., Dudley, Sandra, Nygaard, Sean, Mighell, Taylor L., Grompe, Markus, Harding, Cary O.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6962637/
https://www.ncbi.nlm.nih.gov/pubmed/31970201
http://dx.doi.org/10.1016/j.omtm.2019.12.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6962637/
https://www.ncbi.nlm.nih.gov/pubmed/31970201
http://dx.doi.org/10.1016/j.omtm.2019.12.004

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