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The fate of intravenously administered tetrahydrobiopterin and its implications for heterologous gene therapy of phenylketonuria
Tetrahydrobiopterin (BH(4)) is a required cofactor for the enzymatic activity of phenylalanine hydroxylase (PAH) and is synthesized de novo from GTP in several tissues. Heterologous expression of PAH in tissues other than liver is a potential novel therapy for human phenylketonuria that is completel...
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| Auteurs principaux: | , , , , , , |
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| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
2004
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2694038/ https://ncbi.nlm.nih.gov/pubmed/14728991 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2003.10.002 |
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