发明名称 Compositions and methods for inhibiting expression of transthyretin
摘要 The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.
申请公布号 US8741866(B2) 申请公布日期 2014.06.03
申请号 US201213410262 申请日期 2012.03.01
申请人 Alnylam Pharmaceuticals, Inc. 发明人 Sah Dinah Wen-Yee;Hinkle Gregory;Alvarez Rene;Milstein Stuart;Chen Qingmin
分类号 A61K48/00;C12N15/85;C07H21/02;C07H21/04 主分类号 A61K48/00
代理机构 代理人
主权项 1. A method of inhibiting TTR expression in a cell, the method comprising: (a) contacting the cell with a double-stranded ribonucleic acid (dsRNA) for inhibiting expression of transthyretin (TTR), wherein the dsRNA comprises a sense strand and an antisense strand, the antisense strand comprising a region complementary to an mRNA encoding transthyretin (TTR), wherein the region of complementarity comprises SEQ ID NO:170 and each strand of the dsRNA is 19, 20, 21, 22, 23, or 24 nucleotides in length; and (b) maintaining the cell produced in step (a) for a time sufficient to obtain degradation of the mRNA transcript of a TTR gene, thereby inhibiting expression of the TTR gene in the cell.
地址 Cambridge MA US