IP Library Granted Patent US 8,344,126
Granted Patent B2
US 8,344,126 · App. 12/970,189 · Granted Jan 1, 2013

Compositions and methods for inhibiting expression of XBP-1 gene

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Quick Facts
Patent No.
US 8,344,126
App. No.
12/970,189
Granted
Jan 1, 2013
Kind
B2
Abstract

The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting X-Box Protein 1 (XBP-1), and methods of using the dsRNA to inhibit expression of XBP-1.

Claims (14)

1. A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of an X-Box Protein 1 (XBP-1) gene, wherein said dsRNA consists of a sense strand and an antisense strand, wherein the antisense strand consists of the nucleotide sequence of CUUAACUCCUGGUUCUCAA (SEQ ID NO: 2148) and wherein the sense strand consists of the nucleotide sequence of UUGAGAACCAGGAGUUAAG (SEQ ID NO: 2147).

2. The dsRNA of claim 1 , wherein at least one nucleotide of said sense strand or said antisense strand is a modified nucleotide.

3. The dsRNA of claim 2 , wherein said modified nucleotide is chosen from the group of: a 2′-O-methyl modified nucleotide, a nucleotide comprising a 5′-phosphorothioate group, and a terminal nucleotide linked to a cholesteryl derivative, vitamin E, or dodecanoic acid bisdecylamide group.

4. The dsRNA of claim 2 , wherein said modified nucleotide is chosen from the group of: a 2′-deoxy-2′-fluoro modified nucleotide, a 2′-deoxy-modified nucleotide, a locked nucleotide, an abasic nucleotide, 2′-amino-modified nucleotide, 2′-alkyl-modified nucleotide, morpholino nucleotide, a phosphoramidate, and a non-natural base comprising nucleotide.

5. A cell comprising the dsRNA of claim 1 .

6. A pharmaceutical composition for inhibiting expression of an XBP-1 gene comprising the dsRNA of claim 1 .

7. A method of inhibiting XBP-1 expression in a cell, the method comprising:

(a) introducing into the cell the dsRNA of claim 1 ; and

(b) maintaining the cell produced in step (a) for a time sufficient to obtain degradation of the mRNA transcript of the XBP-1 gene, thereby inhibiting expression of the XBP-1 gene in the cell.

8. A vector comprising a regulatory sequence operably linked to the dsRNA of claim 1 .

9. A cell comprising the vector of claim 8 .

10. The dsRNA of claim 1 , wherein the antisense strand consists of SEQ ID NO: 2176.

11. The dsRNA of claim 1 , wherein the sense strand consists of SEQ ID NO: 2175.

12. The dsRNA of claim 1 , wherein said dsRNA inhibits the expression of an XBP-1 gene by at least 40%.

Assignments (1)
SECURITY INTEREST Recorded Oct 1, 2025
From: ALNYLAM PHARMACEUTICALS, INC.; SIRNA THERAPEUTICS, INC.
To: BANK OF AMERICA, N.A.
Reel/Frame 072996/0337 →