NUCLEIC ACID MOLECULES WITH ENHANCED ACTIVITY

    公开(公告)号:EP3353300A1

    公开(公告)日:2018-08-01

    申请号:EP16770006.1

    申请日:2016-09-21

    摘要: Double-stranded nucleic acid molecules (100, 200, 300, 400, 500 and 600) are provided comprising a sense strand (110, 210, 310, 410, 510 and 610) and an antisense strand (120, 220, 320, 420, 520 and 620). The sense strand has a 3′-overhang (115, 215, 315, 415, 515 and 615) of from four to eight nucleotides. The double-stranded nucleic acid molecules include double-stranded RNA (dsRNA), double-stranded DNA (dsDNA), double-stranded DNA-RNA (sense-antisense) hybrid and double-stranded RNA-DNA (sense-antisense) hybrid. The double-stranded nucleic acid molecules have beneficial properties in terms of unaided cellular delivery (e.g. in the absence of a cationic agent), cellular delivery and release of the antisense strand in a cell as a single-stranded molecule and the delivery of the antisense strand into the nucleus of a cell as a single-stranded molecule. Related compositions, cells, uses and methods are also provided.

    METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA
    6.
    发明授权
    METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA 有权
    通过双链RNA特异性抑制基因表达的方法和组合物

    公开(公告)号:EP1742958B1

    公开(公告)日:2017-05-17

    申请号:EP05732148.1

    申请日:2005-03-15

    IPC分类号: C12N15/11 C12N15/113

    摘要: The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA ("dsRNA"), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.

    摘要翻译: 本发明涉及用于选择性地减少细胞中所需靶基因的基因产物表达的组合物和方法,以及治疗由基因表达引起的疾病的组合物和方法。 更具体地说,本发明涉及含有双链RNA(“dsRNA”)的组合物及其制备方法,其能够减少真核细胞中靶基因的表达。 dsRNA具有长度在25至约30个核苷酸之间的第一寡核苷酸序列和在生物学条件下与第一序列退火的第二寡核苷酸序列。 另外,具有至少19个核苷酸的序列长度的dsRNA序列之一的区域与由靶基因产生的RNA的核苷酸序列充分互补以引发RNAi机制破坏靶RNA。