AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

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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Bibliographic Details
Main Authors: Daelyn Y. Richards, Shelley R. Winn, Sandra Dudley, Sean Nygaard, Taylor L. Mighell, Markus Grompe, Cary O. Harding
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
phenylketonuria
phenylalanine
gene therapy
gene editing
gene correction
CRISPR/Cas9
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050119301500

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http://www.sciencedirect.com/science/article/pii/S2329050119301500

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