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 that 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 US2011130443(A1) 申请公布日期 2011.06.02
申请号 US201113010300 申请日期 2011.01.20
申请人 VORNLOCHER HANS-PETER;MARAGANORE JOHN M 发明人 VORNLOCHER HANS-PETER;MARAGANORE JOHN M.
分类号 A61K31/713;A61P7/02;C07H21/04;C12N5/10;C12N15/113;C12N15/63 主分类号 A61K31/713
代理机构 代理人
主权项
地址