Patent ID: 9045754
Filing Date: 2015-06-02
Classification: A61P,C12N,C12Y

Abstract:
1. A method of inhibiting a target nucleid acid in a cell in an animal comprising administering to the animal a compound comprising a short antisense oligonucleotide consisting of 12 to 14 linked nucleosides and having: a gap region consisting of 8 to 12 linked 2′-deoxynucleosides; a 5′-wing region adjacent to the gap region at the 5′ side of the gap region and consisting of 1 to 3 linked modified nucleosides; and a 3′-wing region adjacent to the gap region at the 3′ side of the gap region and consisting of 1 to 3 linked modified nucleosides; wherein at least one modified nucleoside of one or both of the 5′-wing region and the 3′-wing region is a high-affinity modified monomer; and thereby inhibiting the target nucleic acid in the cell in the animal.