发明名称 Compositions and methods for inhibiting expression of the HAMP gene
摘要 The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the HAMP gene (HAMP 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 HAMP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HAMP gene expression and the expression of the HAMP gene using the pharmaceutical composition.
申请公布号 US8791250(B2) 申请公布日期 2014.07.29
申请号 US201313900854 申请日期 2013.05.23
申请人 Alnylam Pharmaceuticals, Inc. 发明人 Nakayama Tomoko;Geick Anke;Tan Pamela;Lin Herbert Y.
分类号 C12N15/11;A61K48/00;C07H21/02;C07H21/04 主分类号 C12N15/11
代理机构 Fenwick & West LLP 代理人 Fenwick & West LLP
主权项 1. A double-stranded ribonucleic acid (dsRNA), wherein the dsRNA comprises an antisense strand and a sense strand, wherein each strand is less than 30 nucleotides in length, and wherein the target sequence of the dsRNA comprises the first nineteen nucleotides of SEQ ID NO:29 (GAACAUAGGUCUUGGAAUA).
地址 Cambridge MA US