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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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Richards, Daelyn Y., Winn, Shelley R., Dudley, Sandra, Nygaard, Sean, Mighell, Taylor L., Grompe, Markus, Harding, Cary O.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6962637/
https://ncbi.nlm.nih.gov/pubmed/31970201
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.12.004
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