Prospects for Cell-Directed Curative Therapy of Phenylketonuria (PKU)
Phenylketonuria (PKU) due to recessively inherited phenylalanine hydroxylase (PAH) deficiency is among the most common inborn errors of metabolism. Dietary therapy begun early in infancy prevents the major manifestations of the disease but shortcomings to treatment continue to exist including lifelo...
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| Format: | Article |
| Language: | English |
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World Scientific Publishing
2019-12-01
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| Series: | Molecular Frontiers Journal |
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| Online Access: | http://www.worldscientific.com/doi/pdf/10.1142/S2529732519400145 |