Compositions and methods for inhibiting expression of a mutant gene
    45.
    发明申请
    Compositions and methods for inhibiting expression of a mutant gene 有权
    用于抑制突变基因表达的组合物和方法

    公开(公告)号:US20050074757A1

    公开(公告)日:2005-04-07

    申请号:US10384463

    申请日:2003-03-07

    摘要: The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a complementary RNA strand having a complementary region that is substantially complementary to a portion of the mutant gene, and which is partially complementary to the corresponding wild-type gene. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.

    摘要翻译: 本发明涉及用于抑制突变基因表达的双链核糖核酸(dsRNA),其包含互补RNA链,其具有与突变基因的一部分基本上互补的互补区,并且其部分互补于 相应的野生型基因。 本发明还涉及包含dsRNA和药学上可接受的载体的药物组合物。 药物组合物可用于抑制靶突变基因的表达,以及用于治疗由靶基因表达引起的疾病。 本发明还涉及抑制靶突变基因表达的方法,以及用于治疗由靶基因表达引起的疾病的方法。