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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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Bibliografiske detaljer
Udgivet i:Mol Front J
Hovedforfatter: Harding, Cary O.
Format: Artigo
Sprog:Inglês
Udgivet: 2019
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7286632/
https://ncbi.nlm.nih.gov/pubmed/32524084
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1142/s2529732519400145
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