发明名称 COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF FACTOR V LEIDEN MUTANT GENE
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.
申请公布号 CA2626690(A1) 申请公布日期 2007.05.18
申请号 CA20062626690 申请日期 2006.11.07
申请人 ALNYLAM PHARMACEUTICALS, INC. 发明人 MARAGANORE, JOHN M.;VORNLOCHER, HANS-PETER
分类号 A61K48/00;C12N15/113 主分类号 A61K48/00
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