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

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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Bibliographic Details
Main Authors: Sofia Stenler, Oscar PB Wiklander, Maria Badal-Tejedor, Janne Turunen, Joel Z Nordin, David Hallengärd, Britta Wahren, Samir EL Andaloussi, Mark W Rutland, CI Edvard Smith, Karin E Lundin, Pontus Blomberg
Format: Article
Language:English
Published: Elsevier 2014-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
nano
RNA therapeutics
RNA editing
pre-mRNA
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116302797

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http://www.sciencedirect.com/science/article/pii/S2162253116302797

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