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
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