发明名称 LIPOPHILIC DERIVATIVES OF DOUBLE-STRANDED RIBONUCLEIC ACID
摘要 The present invention relates to a double-stranded ribonucleic acid (dsRNA) having improved efficiency of inhibition of gene expression, methods of making the dsRNA, and pharmaceutical compositions comprising the dsRNA. The dsRNA comprises an RNA strand (complementary RNA strand) having a region which is complementary to an RNA transcript of at least a part of a target gene, and at least one covalently linked lipophilic group. The dsRNA are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.
申请公布号 WO2004065601(A3) 申请公布日期 2004.11.18
申请号 WO2004US01461 申请日期 2004.01.21
申请人 ALNYLAM EUROPE AG;HADWIGER, PHILIPP;JOHN, MATTHIAS;LORENZ, CHRISTINA;VORNLOCHER, HANS-PETER;KREUTZER, ROLAND;LIMMER, STEFAN;WILLIAMS, KATHLEEN, MADDEN 发明人 HADWIGER, PHILIPP;JOHN, MATTHIAS;LORENZ, CHRISTINA;VORNLOCHER, HANS-PETER;KREUTZER, ROLAND;LIMMER, STEFAN;WILLIAMS, KATHLEEN, MADDEN
分类号 A61K38/00;A61P31/14;C12N15/11;C12N15/113 主分类号 A61K38/00
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