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 |
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代理人 |
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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.
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地址 |
Cambridge MA US |