Title : Long-term correction of murine phenylketonuria by viral gene transfer: liver versus muscle.

Pub. Date : 2010 Dec

PMID : 20151201






2 Functional Relationships(s)
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1 When transgenic PKU mice that constitutively expressed PAH in muscle were given intraperitoneal supplementation with BH(4), this produced (transient) effective clearance of Phe to normal levels. Phenylalanine phenylalanine hydroxylase Mus musculus
2 In addition, use of an AAV vector containing the genes for PAH, and for two key synthetic enzymes for BH(4), provided substantial and long-lasting correction (more than 1 year) of blood Phe levels when injected into skeletal muscle of PKU mice. Phenylalanine phenylalanine hydroxylase Mus musculus