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Modifications to the siRNA guide strand 5ʹ end that improve therapeutic potency

Structure-guided computational screening identified modifications to the guide strand that could improve the therapeutic properties of siRNA therapeutics. Computational analysis of the structure of the siRNA guide strand in complex with argonaute RISC catalytic component 2 (AGO2) showed that incorporation of triazole derivatives into the siRNA guide strand enhanced binding to AGO2. In a human cell line, siRNA that incorporated triazole-based nucleotides into the guide strand 5ʹ end had greater potency than unmodified siRNA with similar stability. Next steps include testing the modifications in therapeutically relevant siRNAs.

SciBX 6(48); doi:10.1038/scibx.2013.1404
Published online Dec. 19, 2013

Unpatented; licensing status not applicable

Onizuka, K. et al. J. Am. Chem. Soc.; published online Oct. 23, 2013;
Contact: Peter A. Beal, University of California, Davis, Calif.
Contact: Dean J. Tantillo, same affiliation as above