Modified siRNAs containing 2'-fluorinated nucleotides

CARB 41

JK. Watts1, F. Robert2, A. Kalota3, J. Pelletier2, JM. García Fernández4, J. Defaye5, A. M. Gewirtz3, and MJ. Damha1. (1) Department of Chemistry, McGill University, Montreal, QC H3A 2K6, Canada, (2) Department of Biochemistry and McGill Cancer Center, McGill University, Montreal, QC H3G 1Y6, Canada, (3) University of Pennsylvania School of Medicine, Philadelphia, PA 19104, (4) Instituto de Investigaciones Quimicas, CSIC, Universidad de Sevilla, Sevilla, E-41092, Saipan, (5) CNRS and Université Joseph-Fournier-Grenoble 1, Meylan, F-38243, France
This presentation will describe recent work on modified siRNA duplexes containing 2'-fluorinated nucleotides, for knockdown of several human and mouse gene targets. Rationally-designed siRNA sense strand architectures allow significant improvements in potency while retaining a high percentage (>50%) of modified nucleotides. New delivery vehicles will be described and compared with existing transfection technologies.