Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter

Limited duration of transgene expression, insertional mutagenesis, and size limitations for transgene cassettes pose challenges and risk factors for many gene therapy vectors. Here, we report on physiological expression of liver phenylalanine hydroxylase (PAH) by delivery of naked DNA/minicircle (MC...

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Dettagli Bibliografici
Pubblicato in:Mol Ther Nucleic Acids
Autori principali: Grisch-Chan, Hiu Man, Schlegel, Andrea, Scherer, Tanja, Allegri, Gabriella, Heidelberger, Raphael, Tsikrika, Panagiota, Schmeer, Marco, Schleef, Martin, Harding, Cary O., Häberle, Johannes, Thöny, Beat
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2017
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5423318/
https://ncbi.nlm.nih.gov/pubmed/28624210
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.04.013
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