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Expression of phenylalanine hydroxylase (PAH) in erythrogenic bone marrow does not correct hyperphenylalaninemia in Pah(enu2) mice

BACKGROUND: Treatment of many inherited liver enzyme deficiencies requires the removal of toxic intermediate metabolites from the blood of affected individuals. We propose that circulating toxins can be adequately cleared and disease phenotype influenced by enzyme expressed in tissues other than the...

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Bibliographische Detailangaben
Hauptverfasser: Harding, Cary O., Neff, Mark, Jones, Kelly, Wild, Krzysztof, Wolff, Jon A.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2003
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2694059/
https://ncbi.nlm.nih.gov/pubmed/14601136
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jgm.432
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