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COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF FACTOR V LEIDEN MUTANT GENE

机译:抑制因子V LEIDEN突变基因表达的组合物和方法

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.
机译:本发明涉及一种用于抑制因子V Leiden突变基因(因子V Leiden突变基因)的表达的双链核糖核酸(dsRNA),其包含具有小于25个核苷酸长度的核苷酸序列的反义链,并且与因子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和药学上可接受的载体的药物组合物。使用该药物组合物治疗由因子V Leiden突变基因的表达引起的疾病的方法;和抑制因子V Leiden突变基因在细胞中表达的方法。

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