发明名称 |
Compositions and methods for inhibiting expression of a gene from the JC virus |
摘要 |
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the JC Virus (JC virus genome), 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 a gene from the JC Virus. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by JC virus expression and the expression of a gene from the JC Virus using the pharmaceutical composition; and methods for inhibiting the expression of a gene from the JC Virus in a cell. |
申请公布号 |
US9012624(B2) |
申请公布日期 |
2015.04.21 |
申请号 |
US201313779116 |
申请日期 |
2013.02.27 |
申请人 |
Alnylam Pharmaceuticals, Inc. |
发明人 |
Tan Pamela;Sah Dinah;Bramlage Birgit |
分类号 |
C07H21/02;C12N15/11;A61K48/00 |
主分类号 |
C07H21/02 |
代理机构 |
Fenwick & West LLP |
代理人 |
Fenwick & West LLP |
主权项 |
1. A double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a human JC virus genome in a cell, wherein said dsRNA comprises a sense strand and an antisense strand, the antisense strand comprising at least 16 contiguous nucleotides of the nucleotide sequence of SEQ ID NO: 330, wherein a region of complementarity between the antisense strand and a target sequence is less than 30 nucleotides in length and wherein said dsRNA, upon contact with a cell expressing said JC virus, inhibits expression of said JC virus genome. |
地址 |
Cambridge MA US |