A porcine model of phenylketonuria generated by CRISPR/Cas9 genome editing

Author:

Koppes Erik A.ORCID,Redel Bethany K.,Johnson Marie A.,Skvorak Kristen J.,Ghaloul-Gonzalez Lina,Yates Megan E.ORCID,Lewis Dale W.,Gollin Susanne M.ORCID,Wu Yijen L.ORCID,Christ Shawn E.ORCID,Yerle Martine,Leshinski Angela,Spate Lee D.,Benne Joshua A.,Murphy Stephanie L.,Samuel Melissa S.,Walters Eric M.ORCID,Hansen Sarah A.,Wells Kevin D.ORCID,Lichter-Konecki Uta,Wagner Robert A.,Newsome Joseph T.,Dobrowolski Steven F.,Vockley Jerry,Prather Randall S.ORCID,Nicholls Robert D.ORCID

Funder

NIH/NINDS

NIH/NIAID

Publisher

American Society for Clinical Investigation

Subject

General Medicine

Reference78 articles.

1. Donlon J, Sarkissian C, Levy H, Scriver CR. Hyperphenylalaninemia: phenylalanine hydroxylase deficiency. In: Valle D, Antonarakis S, Ballabio A, Beaudet A, Mitchell GA, eds. The Online Metabolic and Molecular Bases of Inherited Disease. McGraw-Hill; Accessed August 10, 2020. https://ommbid-mhmedical-com.pitt.idm.oclc.org/content.aspx?bookid=2709§ionid=225081923

2. Phenylalanine hydroxylase deficiency: diagnosis and management guideline

3. PAHdb: A locus-specific knowledgebase

4. Phenylketonuria: an inborn error of phenylalanine metabolism;Williams;Clin Biochem Rev,2008

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