发明名称 |
COMPOSITIONS AND METHODS FOR INHIBITION OF EXPRESSION OF PROTEIN C (PROC) GENES |
摘要 |
The invention relates to double-stranded ribonucleic acid (dsRNA) targeting a PROC gene, and methods of using the dsRNA to inhibit expression of PROC. At least one nucleotide of the dsRNA can be a modified nucleotide, e.g., a 2-0-methyl modified nucleotide, a nucleotide comprising a 5′-phosphorothioate group, and a terminal nucleotide linked to a cholesteryl derivative or dodecanoic acid bisdecylamide group. Other examples of modified nucleotides include a 2′-deoxy-2′-fluoro modified nucleotide, a 2′-deoxymodified 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. A dsRNA of the invention can include one or more of any of these modified nucleotides. |
申请公布号 |
EP2723351(A4) |
申请公布日期 |
2015.04.22 |
申请号 |
EP20120802782 |
申请日期 |
2012.06.21 |
申请人 |
ALNYLAM PHARMACEUTICALS, INC. |
发明人 |
TOUDJARSKA, IVANKA;MARAGANORE, JOHN, M.;BETTENCOURT, BRIAN;MILSTEIN, STUART;MAIER, MARTIN;CHARISSE, KLAUS;RAJEEV, KALLANTHOTTATHIL;KUCHIMANCHI, SATYANARAYANA |
分类号 |
A61K31/713;C12N15/113 |
主分类号 |
A61K31/713 |
代理机构 |
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代理人 |
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