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Nucleic Acids Symposium Series 2008 52(1):45; doi:10.1093/nass/nrn023
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© 2008 Oxford University Press

This article appears in the following Nucleic Acid Symposium Series issue: Joint Symposium of the 18th International Roundtable on Nucleosides, Nucleotides and Nucleic Acids and the 35th International Symposium on Nucleic Acids Chemistry [View the issue table of contents]

Potent single stranded RNA inhibition

Troels Koch

Santaris Pharma A/S, Bøge Allé 3, 2970 Hørsholm, Denmark

Abstract

The high affinity provided by LNA is the basis for its high antisense potency. This property offers furthermore the opportunity to make shorter than usual antisense oligonucleotides exhibiting very high in vivo potency and efficacy. The potency of short-stranded LNA is comparable to the very best lipid formulated siRNA's. But in contrast to siRNA short-stranded LNA provides a common platform for highly potent mRNA and microRNA inhibitors.

All these features of LNA will be comprehensively illustrated in the presentation. Inhibition of coding RNA will be illustrated using ApoB-100 as the target, and inhibition of miR-122 will be used to illustrate inhibition of non-coding RNA. The conclusions in the presentation are all based on comprehensive rodent and non-human primate data.


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