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Metabolic engineering as therapy for inborn errors of metabolism – development of mice with phenylalanine hydroxylase expression in muscle

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 liver. Phen...

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Bibliografiske detaljer
Main Authors: Harding, CO, Wild, K, Chang, D, Messing, A, Wolff, JA
Format: Artigo
Sprog:Inglês
Udgivet: 1998
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2694047/
https://ncbi.nlm.nih.gov/pubmed/9797873
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/sj.gt.3300653
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