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Beschreibung

Simplicon is a novel system to effect immediate high sustained protein expression of the target gene(s) without the risk of integration. The technology employs a self-replicating RNA based on the Venezuelan equine encephalitis (VEE) genome. By nature of a synthetic and self-replicative RNA, interferon (IFN) responses occur at the transfection and replication of Simplicon RNA. The IFN responses reduce the expression and lifetime of Simplicon RNA. Therefore, it is important to suppress the IFN responses in Simplicon System. B18R acts as a decoy receptor for Type I Interferons and inhibit IFN-alpha, beta, and omega and other Type I IFN receptors. When only employing B18R, some IFN-beta response still occurs. The vaccinia virus E3L gene encodes two double-stranded RNA binding proteins that suppressed IFN-beta response and improved the expression of Simplicon RNA at RNA transfection and continuous cell culture. In product B18R-E3L plasmid (human codon optimized for B18R and E3L), B18R and E3L are provided as a polycistronic DNA that can be transcribed into a polycistronic mRNA to be co-transfected with Simplicon RNA(s) for IFN suppression. For continuous expression, B18R protein is needed to supply with B18R-conditioned medium (B18R-CM) or recombinant protein.

Strukturformel

SAF-SCR727

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