Micro-minicircle Gene Therapy: Implications of Size on Fermentation, Complexation, Shearing Resistance, and Expression

The minicircle (MC), composed of eukaryotic sequences only, is an interesting approach to increase the safety and efficiency of plasmid-based vectors for gene therapy. In this paper, we investigate micro-MC (miMC) vectors encoding small regulatory RNA. We use a construct encoding a splice-correcting...

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Autors principals: Stenler, Sofia, Wiklander, Oscar PB, Badal-Tejedor, Maria, Turunen, Janne, Nordin, Joel Z, Hallengärd, David, Wahren, Britta, Andaloussi, Samir EL, Rutland, Mark W, Smith, CI Edvard, Lundin, Karin E, Blomberg, Pontus
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2014
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3910003/
https://ncbi.nlm.nih.gov/pubmed/24399204
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2013.67
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