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 and method for treating diseases caused by PCSK9 gene expression. |
申请公布号 |
US2014350075(A1) |
申请公布日期 |
2014.11.27 |
申请号 |
US201414330923 |
申请日期 |
2014.07.14 |
申请人 |
Alnylam Pharmaceuticals, Inc. |
发明人 |
Tan Pamela;Bramlage Birgit;Frank-Kamenetsky Maria;Fitzgerald Kevin;Akinc Akin;Kotelianski Victor |
分类号 |
C12N15/113 |
主分类号 |
C12N15/113 |
代理机构 |
|
代理人 |
|
主权项 |
1. A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of a human proprotein convertase subtilisin kexin 9 (PCSK9) gene in a cell, wherein the dsRNA comprises a sense strand and an antisense strand complementary to at least 15 contiguous nucleotides of a PCSK9 gene and comprises a duplex structure between 15 and 30 base pairs in length. |
地址 |
Cambridge MA US |