Small Interfering RNA Specific For HCV And Therapeutic Agent For Hepatitis C Comprising The Same
    3.
    发明申请
    Small Interfering RNA Specific For HCV And Therapeutic Agent For Hepatitis C Comprising The Same 审中-公开
    丙型肝炎病毒的特异性小干扰RNA和丙型肝炎的治疗剂

    公开(公告)号:US20090318531A1

    公开(公告)日:2009-12-24

    申请号:US11915975

    申请日:2006-05-30

    IPC分类号: A61K48/00 C07H21/02 C12N15/63

    摘要: The present invention relates to a therapeutic reagent for hepatitis C comprising HCV specific short interfering RNA (siRNA) as an effective ingredient. The siRNA of the invention is a double-stranded RNA specific for the nucleotide sequence of HCV which induces viral RNA degradation in mammalian cells and thereby inhibits HCV protein expression and replication. The method of the invention, which includes the step of administrating the synthetic siRNA or a DNA vector encoding the RNA, is thus effective for the treatment of HCV carrier by inhibiting HCV gene expression and replication.

    摘要翻译: 本发明涉及包含HCV特异性短干扰RNA(siRNA)作为有效成分的丙型肝炎治疗试剂。 本发明的siRNA是HCV的核苷酸序列特异性的双链RNA,其在哺乳动物细胞中诱导病毒RNA降解,从而抑制HCV蛋白的表达和复制。 包括给予合成siRNA或编码RNA的DNA载体的步骤的本发明的方法因此通过抑制HCV基因表达和复制而对HCV载体的治疗是有效的。

    Liposome for liver-specific delivery and release of therapeutic nucleic acids or drugs
    4.
    发明授权
    Liposome for liver-specific delivery and release of therapeutic nucleic acids or drugs 有权
    用于肝特异性递送和释放治疗性核酸或药物的脂质体

    公开(公告)号:US08318199B2

    公开(公告)日:2012-11-27

    申请号:US12791600

    申请日:2010-06-01

    IPC分类号: A61K9/127 C12N15/88 A61K48/00

    CPC分类号: A61K31/7088 A61K9/1272

    摘要: Disclosed herein is a composite of a nanoscale particle size. The composite is able to specifically deliver therapeutic agents such as therapeutic nucleic acids or drugs to the liver and selectively release them into hepatic cells to manifest potent therapeutic effects of the therapeutic agents. The composite may be comprised of an apolipoprotein A-1 and a liposome-forming material. A composition containing the composite and a pharmaceutically acceptable carrier is disclosed.

    摘要翻译: 本文公开了纳米级粒度的复合材料。 复合材料能够将治疗剂例如治疗性核酸或药物特异性递送至肝脏并选择性地将其释放到肝细胞中以显示治疗剂的有效治疗作用。 复合材料可以由载脂蛋白A-1和脂质体形成材料组成。 公开了含有该复合物和药学上可接受的载体的组合物。

    LINEAR DOUBLE-STRANDED RNA MOLECULE INTERFERING WITH DIFFERENT TARGET GENES
    7.
    发明申请
    LINEAR DOUBLE-STRANDED RNA MOLECULE INTERFERING WITH DIFFERENT TARGET GENES 审中-公开
    线性双链RNA分子与不同目标基因的干扰

    公开(公告)号:US20110008885A1

    公开(公告)日:2011-01-13

    申请号:US12667738

    申请日:2008-07-07

    IPC分类号: C12N5/071 C07H21/02 C12N15/63

    摘要: A linear double-stranded RNA molecule, which comprises two or more consecutively or convergently linked short interfering RNAs (siRNAs) each reducing the expression of one of different target genes, and a recombinant expression vector comprising double-stranded DNA sequence expressing the linear double-stranded RNA molecule are provided. The linear double-stranded RNA molecule or the recombinant expression vector is useful for a method of reducing expression of target genes in a cell, the method comprising introducing the linear double-stranded RNA molecule or the recombinant expression vector into the cell, whereby the encoded siRNAs target different genes and reduce expression of the target genes. It was also proved that effective gene silencing activity can be induced when each siRNA unit within the linear double-stranded RNA molecule has 18 to 24 nucleotides and, additionally, the gene silencing activity is not affected by inverted orientation of an siRNA.

    摘要翻译: 线性双链RNA分子,其包含两个或更多个连续或趋向连接的短干扰RNA(siRNA),每个短干扰RNA减少一个不同靶基因的表达;以及重组表达载体,其包含表达线性双链RNA的双链DNA序列, 提供了双链RNA分子。 线性双链RNA分子或重组表达载体可用于减少细胞中靶基因表达的方法,该方法包括将线性双链RNA分子或重组表达载体引入细胞,由此编码 siRNA靶向不同的基因并减少靶基因的表达。 还证明当线性双链RNA分子内的每个siRNA单元具有18至24个核苷酸时,可以诱导有效的基因沉默活性,此外,基因沉默活性不受siRNA反向取向的影响。