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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Опубликовано в: :PLoS One
Главные авторы: Bell, Sean M., Wendt, Dan J., Zhang, Yanhong, Taylor, Timothy W., Long, Shinong, Tsuruda, Laurie, Zhao, Bin, Laipis, Phillip, Fitzpatrick, Paul A.
Формат: Artigo
Язык:Inglês
Опубликовано: Public Library of Science 2017
Предметы:
Research Article
Online-ссылка: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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