发明名称 COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE PCSK9 GENE
摘要 The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the PCSK9 gene (PCSK9 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by PCSK9 gene expression and the expression of the PCSK9 gene using the pharmaceutical composition; and -I ----- < fn 1: -. CL K< 0 __5 ------------------ ---- ---- --- ----- E~EIEcE rz, cn U- C ____ I 2 S*g % 111dV__6)iS~
申请公布号 AU2016203687(A1) 申请公布日期 2016.06.23
申请号 AU20160203687 申请日期 2016.06.02
申请人 ALNYLAM PHARMACEUTICALS, INC. 发明人 TAN, PAMELA;BRAMLAGE, BIRGIT;FRANK-KAMENETSKY, MARIA;FITZGERALD, KEVIN;AKINC, AKIN;KOTELIANSKI, VICTOR E.
分类号 C12N15/113;A61K31/713 主分类号 C12N15/113
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