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