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Generated: August 19, 2019

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Claims for Patent: 9,518,259

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Summary for Patent: 9,518,259
Title:Compounds and methods for modulating interaction between proteins and target nucleic acids
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.
Inventor(s): Rigo; Frank (Carlsbad, CA), Bennett; C. Frank (Carlsbad, CA), Krainer; Adrian R. (Huntington Station, NY), Hua; Yimin (Jericho, NY)
Assignee: Ionis Pharmaceuticals, Inc. (Carlsbad, CA)
Application Number:13/703,322
Patent Claims: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.

2. The method of claim 1, wherein the antisense compound binds to the target pre-mRNA and recruits at least one non-cleaving nucleic acid binding protein to the antisense compound/target pre-mRNA duplex.

3. The method of claim 2, wherein at least one of the at least one non-cleaving nucleic acid binding proteins recruited to the compound/target pre-mRNA duplex is Interleukin Enhancer Binding Factor 2 or Interleukin Enhancer Binding Factor 3.

4. The method of claim 1, wherein the target pre-mRNA is associated with a disease or disorder.

5. The method of claim 4, wherein the target pre-mRNA is Bcl-x.

6. The method of claim 4, wherein the disease or disorder is cancer.

7. The method of claim 4, wherein the disease or disorder is Duchenne muscular dystrophy.

8. The method of claim 1, wherein the cell is in vitro.

9. The method of claim 1, wherein the cell is in an animal.

10. The method of claim 1 comprising performing an assay to determine whether the exon has been skipped.

11. The method of claim 1 comprising performing an assay to determine whether a protein has been recruited to the antisense compound/target pre-mRNA duplex.

12. The method of claim 11 comprising performing an assay to determine the identity of one or more protein recruited to the antisense compound/target pre-mRNA duplex.

13. The method of claim 6, wherein the cell is in an animal.

14. The method of claim 7, wherein the cell is in an animal.

Summary for Patent:   Try a Free Trial

PCT Information
PCT FiledJune 15, 2011PCT Application Number:PCT/US2011/040581
PCT Publication Date:December 22, 2011PCT Publication Number:WO2011/159836

Details for Patent 9,518,259

Applicant Tradename Biologic Ingredient Dosage Form BLA Number Approval Date Patent No. Assignee Estimated Patent Expiration Status Orphan Source
Schering INTRON A interferon alfa-2b VIAL 103132 001 1986-06-04   Try a Free Trial Ionis Pharmaceuticals, Inc. (Carlsbad, CA) 2030-06-15 RX search
Schering INTRON A interferon alfa-2b VIAL 103132 002 1986-06-04   Try a Free Trial Ionis Pharmaceuticals, Inc. (Carlsbad, CA) 2030-06-15 RX search
Schering INTRON A interferon alfa-2b VIAL 103132 003 1986-06-04   Try a Free Trial Ionis Pharmaceuticals, Inc. (Carlsbad, CA) 2030-06-15 RX search
Applicant Tradename Biologic Ingredient Dosage Form BLA Number Approval Date Patent No. Assignee Estimated Patent Expiration Status Orphan Source

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