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Article

Metabolic basis of sexual dimorphism in PKU mice after genome-targeted PAH gene therapy.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jun 2007

Chen Li, Thung Swan N, Woo Savio L C

Abstract excerpt

We have previously reported a transgene delivery system based on phiBT1 bacteriophage integrase that results in targeted insertion of transgenes into mammalian genomes, and its use in the delivery of murine phenylalanine hydroxylase (PAH) complementary DNA (cDNA) into the hepatocytes of male phenylketonuria (PKU) mice, leading to a complete and permanent correction of their hyperphenylalaninemic phenotype. In...

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Identifiers

PMID: 17406346
DOI: 10.1038/sj.mt.6300137

Genes

PAH

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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