发明名称 MODIFIED RNAI AGENTS
摘要 One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene. The dsRNA duplex comprises one or more motifs of three identical modifications on three consecutive nucleotides in one or both strand, particularly at or near the cleavage site of the strand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
申请公布号 KR20150021489(A) 申请公布日期 2015.03.02
申请号 KR20147016494 申请日期 2012.11.16
申请人 ALNYLAM PHARMACEUTICALS, INC. 发明人 RAJEEV KALLANTHOTTATHIL G.;ZIMMERMANN TRACY;MANOHARAN MUTHIAH;MAIER MARTIN;KUCHIMANCHI SATYANARAYANA;CHARISSE KLAUS
分类号 C12N15/113;A61K31/7125;A61K31/713 主分类号 C12N15/113
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