Treatment of Phenylketonuria Using Minicircle-Based Naked-DNA Gene Transfer to Murine Liver

Host immune response to viral vectors, persistence of nonintegrating vectors, and sustained transgene expression are among the major challenges in gene therapy. To overcome these hurdles, we successfully used minicircle (MC) naked-DNA vectors devoid of any viral or bacterial sequences for the long-t...

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Publicado en:Hepatology
Main Authors: Viecelli, Hiu Man, Harbottle, Richard P., Wong, Suet Ping, Schlegel, Andrea, Chuah, Marinee K., Vanden Driessche, Thierry, Harding, Cary O., Thöny, Beat
Formato: Artigo
Idioma:Inglês
Publicado: 2014
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4449723/
https://ncbi.nlm.nih.gov/pubmed/24585515
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/hep.27104
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