AUMsaver™

RNase H-Mediated Knockdown: AUMsaver ASOs are single-stranded DNA/RNA-mimicking oligonucleotides, similar in principle to our other products, that bind to target RNA and induce RNase H1 to cleave it. They are typically designed with partial chemical modification (phosphorothioate backbone, maybe some 2′ modifications for stability, but perhaps not fully modified at every position in order to reduce synthesis cost). Once delivered into the cell (via a transfection agent), they localize to the nucleus and cytoplasm and seek out the target RNA. The mode of action is the same: formation of an ASO:RNA duplex -> RNase H cleavage of RNA -> decreased target gene expression.

Requirement for Delivery Reagent: Unlike our self-delivering line, AUMsaver oligos do not penetrate cells efficiently on their own. Therefore, you will use standard transfection reagents or physical methods to introduce them. The AUMsaver ASO is mixed with a lipid nanoparticle or other carrier that facilitates its entry into cells. Once inside, however, no further differences exist – it acts just as any AUM ASO would, by binding RNA.

Design and AI Optimization: AUMsaver ASOs are still designed with our AI platform to provide optimal targeting of your gene of interest. They maintain a phosphorothioated backbone to resist nucleases and promote some gymnotic uptake, giving them an edge in stability over unmodified oligos. They are optimized to strike a balance: high activity, lower synthesis cost.