Formulation and PEGylation optimization of the therapeutic PEGylated phenylalanine ammonia lyase for the treatment of phenylketonuria

Phenylketonuria (PKU) is a genetic metabolic disease in which the decrease or loss of phenylalanine hydroxylase (PAH) activity results in elevated, neurotoxic levels of phenylalanine (Phe). Due to many obstacles, PAH enzyme replacement therapy is not currently an option. Treatment of PKU with an alt...

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Udgivet i:PLoS One
Main Authors: Bell, Sean M., Wendt, Dan J., Zhang, Yanhong, Taylor, Timothy W., Long, Shinong, Tsuruda, Laurie, Zhao, Bin, Laipis, Phillip, Fitzpatrick, Paul A.
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2017
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5345807/
https://ncbi.nlm.nih.gov/pubmed/28282402
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0173269
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