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    DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION
    6.
    发明公开
    DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION 有权
    包含差异修饰的STRANDS的双链组合物用于基因调节

    公开(公告)号：EP2173358A1

    公开(公告)日：2010-04-14

    申请号：EP08771759.1

    申请日：2008-06-23

    申请人： Isis Pharmaceuticals, Inc.

    发明人： BHAT, Balkrishen ,  PRAKASH, Thazha, P. ,  ALLERSON, Charles ,  KINBERGER, Garth, A. ,  MARCUSSON, Eric, G. ,  SWAYZE, Eric, E.

    IPC分类号： A61K31/70 ,  C12N15/00

    CPC分类号： C12N15/111 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3515 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2320/51 ,  C12Y301/03048 ,  C12N2310/3521

    摘要： The present invention provides double stranded compositions wherein the first strand is modified to have a particular motif and the second strand is modified a selected motif. The motifs are defined by positioning of differentially modified nucleosides wherein at least the sugar moieties are different. More particularly, the present compositions comprise an antisense strand that is modified to have a positional/full motif and the sense strand is modified to have an alternating motif, a hemimer motif, a blockmer motif, a gapped motif, a positional motif, a positional/full motif or a fully modified motif. Each strand further comprises one or more phosphorothioate internucleoside linkage. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.

    摘要翻译： 本发明提供了双链组合物，其中第一条链被修饰成具有特定基序并且第二条链被修饰成选定基序。 基序是通过定位差异修饰的核苷定义的，其中至少糖部分是不同的。 更特别地，本发明的组合物包含经修饰以具有位置/完整基序并且有义链经修饰以具有交替基序，半个体基元，封闭剂基序，缺口基序，位置基序，位置 /完整主题或完全修改的主题。 每条链还包含一个或多个硫代磷酸酯核苷间键。 该组合物可用于靶向选定的核酸分子并调节一种或多种基因的表达。 在优选的实施方案中，本发明的组合物与靶RNA的一部分杂交，导致靶RNA的正常功能丧失。 本发明还提供了调节基因表达的方法。