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    • 6. 发明授权
    • Compositions and methods for inhibiting expression of a mutant gene
    • 用于抑制突变基因表达的组合物和方法
    • US08273870B2
    • 2012-09-25
    • US13177316
    • 2011-07-06
    • Roland KreutzerStefan LimmerMatthias John
    • Roland KreutzerStefan LimmerMatthias John
    • C07H21/04
    • C12N15/1131C12N15/111C12N2310/14C12N2310/53C12N2320/50
    • The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a first strand that has a complementary region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand. 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.
    • 本发明涉及用于抑制突变基因表达的双链核糖核酸(dsRNA),其包含第一链,该第一链具有与至少部分至少部分的RNA转录物的至少一部分互补的互补区 突变靶基因和与第一链互补或基本上互补的dsRNA的第二链。 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。