Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
    39.
    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:US08658356B2

    公开(公告)日:2014-02-25

    申请号:US13152686

    申请日:2011-06-03

    IPC分类号: C12Q1/68

    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降低来自细胞中所需靶基因的基因产物的表达以及用于治疗由基因表达引起的疾病的组合物和方法。 更具体地,本发明涉及含有双链RNA(“dsRNA”)的组合物及其制备方法,其能够降低真核细胞中靶基因的表达。 dsRNA具有长度为25至约30个核苷酸的第一寡核苷酸序列和在生物条件下与第一序列退火的第二寡核苷酸序列。 此外,具有至少19个核苷酸的序列长度的dsRNA的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,以通过RNAi机制触发靶RNA的破坏。