DOUBLE-STRANDED LIPID-MODIFIED RNA HAVING HIGH RNA INTERFERENCE EFFECT
    2.
    发明公开
    DOUBLE-STRANDED LIPID-MODIFIED RNA HAVING HIGH RNA INTERFERENCE EFFECT 有权
    DOPPELSTRÄNGIGE脂质变性RNA麻醉HOHEM RNA-INTERFERENZEFFEKT

    公开(公告)号:EP2264167A1

    公开(公告)日:2010-12-22

    申请号:EP09729165.2

    申请日:2009-03-31

    摘要: An object of the present invention is to provide a novel double-stranded RNA that has high resistance to nuclease and cellular uptake efficiency and that can produce an excellent RNA interference effect. The present invention provides a double-stranded lipid-modified RNA comprising a sense strand having a nucleotide sequence complementary to a target sequence in a target gene, and an antisense strand having a nucleotide sequence complementary to the sense strand, the double-stranded RNA being capable of suppressing the expression of the target gene, and the sense strand having a double-stranded lipid bound directly or via a linker to at least one of the first to sixth nucleotides from the 5' end.

    摘要翻译: 本发明的目的是提供对核酸酶和细胞摄取效率具有高抗性并且可以产生优异的RNA干扰效果的新型双链RNA。 本发明提供了包含具有与靶基因中的靶序列互补的核苷酸序列的有义链和具有与有义链互补的核苷酸序列的反义链的双链脂质修饰的RNA,所述双链RNA为 能够抑制靶基因的表达,并且具有直链或经由连接体结合至5'末端的至少一个第一至第六个核苷酸的双链脂质的有义链。

    COMPLEX OF POLYSACCHARIDE AND DOUBLE-STRANDED RNA
    4.
    发明公开
    COMPLEX OF POLYSACCHARIDE AND DOUBLE-STRANDED RNA 有权
    KOMPLEX AUS POLYSACCHARID UND DOPPELSTRANG-RNA

    公开(公告)号:EP2226384A1

    公开(公告)日:2010-09-08

    申请号:EP08862702.1

    申请日:2008-12-18

    IPC分类号: C12N15/09

    摘要: An object of the present invention is to provide novel double-stranded RNA having an RNA interference effect, in which the cellular uptake and the resistance to enzymatic degradation are improved, without reducing the RNA interference effect.
    The cellular uptake and the resistance to enzymatic degradation of a complex of a polysaccharide having a β-1,3-glucan skeleton and double-stranded RNA can be significantly improved while maintaining the RNA interference effect, by fulfilling the following conditions (i) to (iii): (i) the double-stranded RNA has a sense strand consisting of a base sequence complementary to a target sequence in a target gene and an antisense strand containing a base sequence complementary to the sense strand, and the double-stranded RNA can inhibit expression of the target gene; (ii) the double-stranded RNA has a single-stranded polydeoxyadenine bound directly or via a linker to the end of at least one of the sense strand and the antisense strand; and (iii) the polysaccharide and the single-stranded polydeoxyadenine form a complex.

    摘要翻译: 本发明的目的是提供具有RNA干扰作用的新型双链RNA,其中细胞摄取和对酶降解的抗性得到改善,而不降低RNA干扰效应。 通过满足以下条件,可以显着改善具有2-1,3-葡聚糖骨架和双链RNA的多糖的复合物的细胞摄取和耐酶降解,同时保持RNA干扰效应(i)至 (iii):(i)双链RNA具有由与靶基因中的靶序列互补的碱基序列的有义链和含有与有义链互补的碱基序列的反义链,双链RNA 可抑制靶基因的表达; (ii)双链RNA具有单链多脱氧腺嘌呤,其直接​​或经由接头连接至至少一个有义链和反义链的末端; 和(iii)多糖和单链多脱氧腺嘌呤形成复合物。