COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF HUNTINGTIN GENE
First Claim
1. A pharmaceutical composition comprising a double stranded ribonucleic acid (dsRNA) comprising a sense strand and an antisense strand and a pharmaceutically acceptable carrier, antisense strand comprises a nucleotide sequence consisting of SEQ ID NO:
- 793 and a 2′
-O-methyl modified nucleotide at location 4 and the sense strand comprises a nucleotide sequence consisting of SEQ ID NO;
792 and a 2′
-O-methyl modified nucleotide at locations 2, 3, 5, 12, 13, 15, and 17.
4 Assignments
0 Petitions
Accused Products
Abstract
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
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Citations
42 Claims
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1. A pharmaceutical composition comprising a double stranded ribonucleic acid (dsRNA) comprising a sense strand and an antisense strand and a pharmaceutically acceptable carrier, antisense strand comprises a nucleotide sequence consisting of SEQ ID NO:
- 793 and a 2′
-O-methyl modified nucleotide at location 4 and the sense strand comprises a nucleotide sequence consisting of SEQ ID NO;
792 and a 2′
-O-methyl modified nucleotide at locations 2, 3, 5, 12, 13, 15, and 17. - View Dependent Claims (11, 12, 13, 16, 17, 18, 19, 20, 21, 22, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36, 37, 38, 39, 40)
- 793 and a 2′
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2. (canceled)
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3. (canceled)
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4. (canceled)
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5. (canceled
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6. (canceled)
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7. (canceled)
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8. (canceled)
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9. (canceled)
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10. (canceled)
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14. (canceled)
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15. (canceled)
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23. (canceled)
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24. (canceled)
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41. A method of inhibiting expression of Huntingtin (HD) gene in a cell in a patient comprising administering to the brain of the patient by intrastriatal infusion a therapeutically effective amount of a pharmaceutical composition comprising a dsRNA and an aqueous buffer, the dsRNA consisting of a sense strand and antisense strand, wherein the antisense strand comprises a nucleotide sequence consisting of SEQ ID N0:
- 793 and a 2′
-O-methyl modified nucleotide at location 4 and a phosphorothioate at the internucleotide linkage between nucleotides 20 and 21 and the sense strand comprises a nucleotide sequence consisting of SEQ ID N0;
792 and said sense strand comprises a 2′
-O-methyl modified nucleotide at locations 2, 3, 5, 12, 13, 15, and 17 and a phosphorothioate at the internucleotide linkage between nucleotides 20 and 21.
- 793 and a 2′
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42. A method of treating or managing Huntintin disease in a patient comprising administering to the brain of the patient by intrastriatal infusion a therapeutically effective amount of a pharmaceutical composition comprising a dsRNA and an aqueous buffer, the dsRNA consisting of a sense strand and antisense strand, wherein the antisense strand comprises a nucleotide sequence consisting of SEQ ID N0:
- 793 and a 2′
-O-methyl modified nucleotide at location 4 and a phosphorothioate at the internucleotide linkage between nucleotides 20 and 21 and the sense strand comprises a nucleotide sequence consisting of SEQ ID N0;
792 and a 2′
-O-methyl modified nucleotide at locations 2, 3, 5, 12, 13, 15, and 17 and a phosphorothioate at the internucleotide linkage between nucleotides 20 and 21.
- 793 and a 2′
Specification