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    COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF FACTOR V LEIDEN MUTANT GENE
    14.
    发明公开
    COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF FACTOR V LEIDEN MUTANT GENE 有权
    ZUSAMMENSETZUNGEN UND VERFAHREN ZUR HEMMUNG DER EXPRESSION VON FAKTOR V LEIDEN MUTATIONSGEN

    公开(公告)号：EP1945270A4

    公开(公告)日：2009-10-21

    申请号：EP06837014

    申请日：2006-11-07

    申请人： ALNYLAM PHARMACEUTICALS INC

    发明人： VORNLOCHER HANS-PETER ,  MARAGANORE JOHN M

    IPC分类号： A61K31/713 ,  C12N15/113

    CPC分类号： C12N15/113 ,  A61K47/554 ,  A61K48/00 ,  C12N2310/14 ,  C12N2320/30

    摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Factor V Leiden mutant gene (Factor V Leiden mutant gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the Factor V Leiden mutant gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the Factor V Leiden mutant gene using the pharmaceutical composition; and methods for inhibiting the expression of the Factor V Leiden mutant gene in a cell.

    摘要翻译： 本发明涉及用于抑制因子V莱顿突变基因（因子V莱顿突变基因）表达的双链核糖核酸（dsRNA），其包含长度小于25个核苷酸的核苷酸序列的反义链，其中 与因子V Leiden突变基因的至少一部分基本上互补。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 用于使用该药物组合物治疗由因子V莱顿突变基因表达引起的疾病的方法; 以及抑制细胞因子V Leiden突变基因表达的方法。