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US 9,670,492 B2Granted
Modulation of prekallikrein (PKK) expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsSusan M. Freier, Huynh-Hoa Bui
Life of this patent
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Abstract
Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.
Claims29
A compound comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides and having a nucleobase sequence comprising a portion of at least 8 contiguous nucleobases 100% complementary to an equal length portion of nucleobases 27427-27466 of SEQ ID NO:
- 10, wherein the nucleobase sequence of the modified oligonucleotide is at least 90% complementary to SEQ ID NO:
- 10 as measured over the entirety of the modified oligonucleotide, wherein the modified oligonucleotide comprises:
- a gap segment consisting of linked deoxynucleosides;
- a 5′ wing segment consisting of linked nucleosides;
- a 3′ wing segment consisting of linked nucleosides;
- wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment and wherein each nucleoside of each wing segment comprises a modified sugar.
3 more elements
A compound consisting of a modified oligonucleotide according to the following formula:
- Tes Ges mCes Aes Aes Gds Tds mCds Tds mCds Tds Tds Gds Gds mCds Aes Aes Aes mCes Ae, wherein,
- A=an adenine,
- mC=a 5-methylcytosine
- G=a guanine,
- T=a thymine,
- e=a 2′-O-methoxyethyl modified nucleoside,
- d=a 2′-deoxynucleoside, and
- s=a phosphorothioate internucleoside linkage.
5 more elements
Dependencies are inferred from the claim text.