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

• Main Authors: Sofia Stenler (Author), Oscar PB Wiklander (Author), Maria Badal-Tejedor (Author), Janne Turunen (Author), Joel Z Nordin (Author), David Hallengärd (Author), Britta Wahren (Author), Samir EL Andaloussi (Author), Mark W Rutland (Author), CI Edvard Smith (Author), Karin E Lundin (Author), Pontus Blomberg (Author)
• Format: Book
• Published: Elsevier, 2014-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...