发明申请
- 专利标题： RNAi Modulation of AHA and Therapeutic Uses Thereof
- 专利标题（中）： AHA的RNAi调节及其治疗用途
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申请号： US12877025申请日： 2010-09-07
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公开(公告)号： US20110003882A1公开(公告)日： 2011-01-06
- 发明人: Rainer Constien , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , William Balch
- 申请人： Rainer Constien , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , William Balch
- 申请人地址： US MA Cambridge
- 专利权人： ALNYLAM PHARMACEUTICALS, INC.
- 当前专利权人： ALNYLAM PHARMACEUTICALS, INC.
- 当前专利权人地址： US MA Cambridge
- 主分类号： A61K31/713
- IPC分类号： A61K31/713 ; C07H21/02 ; A61P35/00 ; A61P11/00 ; A61P1/18 ; C12N5/00 ; C12N15/63
摘要：
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an Aha gene (Aha1 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 an Aha gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Aha1 expression and the expression of an Aha gene using the pharmaceutical composition; and methods for inhibiting the expression of an Aha gene in a cell.
公开/授权文献
- US08114984B2 RNAi modulation of Aha and therapeutic uses thereof 公开/授权日：2012-02-14