Abstract
Phenylketonuria (PKU) is a metabolic disorder secondary to a hepatic deficiency of phenylalanine hydroxylase (PAH) that predisposes affected individuals to the development of severe mental retardation. PAH is the enzyme that catalyzes the conversion of phenylalanine to tyrosine, and its deficiency leads to hyperphenylalaninemia as well as hypopigmentation in patients. We have previously reported a transgene delivery system based on phiBT1 bacteriophage integrase that result in targeted insertion of transgenes into the mammalian genome, and the use of it to transfer a CAG-driven mouse PAH cDNA expression cassett in the hepatocytes of PKU mice.
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