Title : Gene therapy for phenylketonuria.

Pub. Date : 1994 Dec

PMID : 7766948






1 Functional Relationships(s)
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1 In contrast, infusion of a recombinant adenoviral vector expressing the human PAH cDNA into the portal circulation of PAH-deficient mice restores 10-80% of normal hepatic PAH activity and completely normalizes serum phenylalanine levels. Phenylalanine phenylalanine hydroxylase Mus musculus