5′ phosphate mimics
    32.
    发明授权
    5′ phosphate mimics 有权
    5'磷酸酯类似物

    公开(公告)号:US08927513B2

    公开(公告)日:2015-01-06

    申请号:US13382346

    申请日:2010-07-07

    摘要: The present invention provides nucleosides and oligonucleotides comprising a 5′ phosphate mimics of formula (IVc) or (Vc). One aspect of the present invention relates to modified nucleosides and oligonucleotides comprising such dinucleotide of formula (Ia). Another aspect of the invention relates to a method of inhibiting the expression of a gene in call, the method comprising (a) contacting an oligonucleotide of the invention with the cell; and (b) maintaining the cell from step (a) for a time sufficient to obtain degradation of the mRNA of the target gene.

    摘要翻译: 本发明提供了包含式(IVc)或(Vc)的5'磷酸酯模拟物的核苷和寡核苷酸。 本发明的一个方面涉及包含式(Ia)的二核苷酸的修饰核苷和寡核苷酸。 本发明的另一方面涉及一种抑制基因表达的方法,所述方法包括(a)使本发明的寡核苷酸与细胞接触; 和(b)将来自步骤(a)的细胞维持足以获得靶基因的mRNA降解的时间。

    Modified iRNA agents
    38.
    发明授权
    Modified iRNA agents 有权
    修饰的iRNA试剂

    公开(公告)号:US08344125B2

    公开(公告)日:2013-01-01

    申请号:US12619382

    申请日:2009-11-16

    IPC分类号: C07H21/04 A61K31/70

    摘要: The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose. The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a carbohydrate; or a steroid, e.g., cholesterol, which is optionally substituted with at least one carbohydrate. is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents.

    摘要翻译: 本发明涉及iRNA试剂,其优选包括其中核糖部分被除核糖以外的部分替代的单体。 包含这样的单体可以允许调节其引入其中的iRNA试剂的性质,例如通过使用非核糖部分作为配体或其他实体例如碳水化合物的点; 或类固醇,例如胆固醇,其任选被至少一种碳水化合物取代。 是直接或间接地束缚着。 本发明还涉及制备和使用这种修饰的iRNA试剂的方法。