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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)|
|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: Start Trial
|PCT Filed||June 15, 2011||PCT Application Number:||PCT/US2011/040581|
|PCT Publication Date:||December 22, 2011||PCT Publication Number:||WO2011/159836|
|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||Start Trial||Ionis Pharmaceuticals, Inc. (Carlsbad, CA)||2030-06-15||RX||search|
|Schering||INTRON A||interferon alfa-2b||VIAL||103132||002||1986-06-04||Start Trial||Ionis Pharmaceuticals, Inc. (Carlsbad, CA)||2030-06-15||RX||search|
|Schering||INTRON A||interferon alfa-2b||VIAL||103132||003||1986-06-04||Start 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|
|Country||Patent Number||Estimated Expiration|
|World Intellectual Property Organization (WIPO)||2011159836||Start Trial|
|United States of America||2013289092||Start Trial|
|European Patent Office||2582397||Start Trial|
|>Country||>Patent Number||>Estimated Expiration|
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