Complete restoration of phenylalanine ox
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Hiroya Yagi; Tsuyoshi Ogura; Hiroaki Mizukami; Masashi Urabe; Hiromi Hamada; Hir
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Article
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2011
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John Wiley and Sons
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English
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Background Classical phenylketonuria (PKU) arises from a deficiency of phenylalanine hydroxylase (PAH) that catalyses phenylalanine oxidation in the liver. Lack of PAH activity causes massive hyperphenylalaninemia and consequently severe brain damage. Preclinical studies showed that conventional ade