Compounds and methods for modulating interaction between proteins and target nucleic acids
AssigneeIonis Pharmaceuticals, Inc.
InventorsFrank Rigo, C. Frank Bennett, Adrian R. Krainer, Yimin Hua
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Abstract
Provided herein are antisense compounds and methods for recruiting one or more non-cleaving protein to a target nucleic acid in a cell. In certain instances such recruitment of a non-cleaving protein alters the function or activity of the target nucleic acid. In certain such instances, the target nucleic acid a pre-mRNA and the recruitment of the non-cleaving protein results in a change in splicing of the pre-mRNA.
Claims14
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.
Dependencies are inferred from the claim text.