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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Publicat a:Mol Ther Methods Clin Dev
Autors principals: Richards, Daelyn Y., Winn, Shelley R., Dudley, Sandra, Nygaard, Sean, Mighell, Taylor L., Grompe, Markus, Harding, Cary O.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2019
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Article
Accés en línia: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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