Patent ID: 9518259
Filing Date: 2016-12-13
CPC Classification: A61P,C12N

Claim Text:
1. A method of inducing exon skipping in a target pre-mRNA in a cell comprising contacting the cell with an antisense compound comprising a chemically modified oligonucleotide consisting of 18 linked nucleosides; and thereby inducing exon skipping in the pre-mRNA in the cell; wherein the antisense compound comprises: a 5′-wing region consisting of 2 linked duplex stabilizing nucleosides; a 3′-wing region consisting of 2 linked duplex stabilizing nucleosides; and a central gap region located between the 5′-wing region and the 3′-wing region and consisting of 14 contiguous nucleosides comprising 2′-F modifications; wherein each duplex stabilizing nucleoside is a 2′-methoxyethyl (2′-MOE) nucleoside, and wherein the antisense compound is complementary to an intron of the target pre-mRNA.