Low Therapeutic Threshold for Hepatocyte Replacement in Murine Phenylketonuria

Phenylalanine homeostasis in mammals is primarily controlled by liver phenylalanine hydroxylase (PAH) activity. Inherited PAH deficiency (phenylketonuria or PKU) leads to hyperphenylalaninemia in both mice and humans. A low level of residual liver PAH activity ensures near-normal dietary protein tol...

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Main Authors: Hamman, Kelly, Clark, Heather, Montini, Eugenio, Al-Dhalimy, Muhsen, Grompe, Markus, Finegold, Milton, Harding, Cary O.
Format: Artigo
Sprog:Inglês
Udgivet: 2005
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2694052/
https://ncbi.nlm.nih.gov/pubmed/16043102
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2005.03.025
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