Title : Gene therapy for phenylketonuria: phenotypic correction in a genetically deficient mouse model by adenovirus-mediated hepatic gene transfer.

Pub. Date : 1994 Jul

PMID : 7584088






1 Functional Relationships(s)
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1 Although this therapeutic effect did not persist, analysis of the relationship between hepatic PAH activity and serum phenylalanine levels indicated that only 10-20% of normal enzymatic activity in the mouse liver is sufficient to restore normal serum phenylalanine levels. Phenylalanine phenylalanine hydroxylase Mus musculus