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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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Publicat a:Hepatology
Autors principals: Viecelli, Hiu Man, Harbottle, Richard P., Wong, Suet Ping, Schlegel, Andrea, Chuah, Marinee K., Vanden Driessche, Thierry, Harding, Cary O., Thöny, Beat
Format: Artigo
Idioma:Inglês
Publicat: 2014
Matèries:
Accés en línia: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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