Some of the most important desired features in any gene-silencing or manipulation tool are:
(1) Efficient knockdown or regulation of the target RNA.
(2) Ability to bind to the target RNA (mRNA, miRNA, or lncRNA) in a high sequence specific manner.
(3) No toxicity.
(4) Most importantly – efficient delivery without an external source (e.g. without a transfection agent, formulation, conjugate or viral vector).
Antisense oligonucleotides designed using next-generation FANA technology can effectively and efficiently perform all these activities.
Antisense oligonucleotides offer the ability to target and silence specific sequences of RNA. These molecules consist of a short segment of DNA flanked by two segments of RNA, linked with a phosphorothioate backbone. However, they are not without their own set of challenges, and can suffer from a lack of stability and/or affinity for their target. Both of these properties can be improved by the incorporation of a modified sugar in the RNA backbone, which changes the RNA segments to a similar but more effective molecule. This next-generation molecule is called Fluoroarabinonucleic acid, or 2’ F-ANA.