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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Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Mol Ther Nucleic Acids
Päätekijät: 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
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: American Society of Gene & Cell Therapy 2017
Aiheet:
Original Article
Linkit: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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