发明申请
WO2004027030A2 EFFICIENT REDUCTION OF TARGET RNA’S BY SINGLE- AND DOUBLE-STRANDED OLIGOMERIC COMPOUNDS
审中-公开
基本信息:
- 专利标题： EFFICIENT REDUCTION OF TARGET RNA’S BY SINGLE- AND DOUBLE-STRANDED OLIGOMERIC COMPOUNDS
- 专利标题（中）：单链和双链寡聚化合物有效减少目标RNA
- 申请号：PCT/US2003/029294 申请日：2003-09-18
- 公开(公告)号：WO2004027030A2 公开(公告)日：2004-04-01
- 发明人： VICKERS, Timothy , KOO, Seongjoon , BENNETT, Frank, C. , CROOKE, Stanley, T. , DEAN, Nicholas, M. , BAKER, Brenda, F.
- 申请人： ISIS PHARMACEUTICALS, INC. , VICKERS, Timothy , KOO, Seongjoon , BENNETT, Frank, C. , CROOKE, Stanley, T. , DEAN, Nicholas, M. , BAKER, Brenda, F.
- 申请人地址： 2292 Faraday Avenue, Carlsbad, CA 92008 US
- 专利权人： ISIS PHARMACEUTICALS, INC.,VICKERS, Timothy,KOO, Seongjoon,BENNETT, Frank, C.,CROOKE, Stanley, T.,DEAN, Nicholas, M.,BAKER, Brenda, F.
- 当前专利权人： ISIS PHARMACEUTICALS, INC.,VICKERS, Timothy,KOO, Seongjoon,BENNETT, Frank, C.,CROOKE, Stanley, T.,DEAN, Nicholas, M.,BAKER, Brenda, F.
- 当前专利权人地址： 2292 Faraday Avenue, Carlsbad, CA 92008 US
- 代理机构： ATTWELL, Gwilym, John, Owen
- 优先权： US60/411,780 20020918
- 主分类号： C12N
- IPC分类号： C12N
摘要：
The present invention provides, inter alia, methods of selecting a singlestranded oligomeric compounds for inhibiting RNA expression, methods of generating double-stranded oligomeric compounds, methods of identifying optimized double-stranded oligomeric compounds, methods of selecting optimized single-stranded oligomeric compounds, methods of selecting optimized double-stranded oligomeric compounds, methods of identifying multifunctional oligomeric compounds, methods for optimizing target region selection for modulation of RNA expression, methods of optimizing expression modulation of RNA, and the like. The present invention further provides oligomeric compounds, 8-80 nucleobases in length targeted to a target RNA, wherein said oligomeric compound hybridizes to said target RNA and inhibits RNA levels by at least 50% in both single-stranded and double-stranded forms, and multifunctional oligomeric compounds.
摘要（中）：
本发明尤其提供了选择用于抑制RNA表达的单链寡聚化合物的方法，产生双链寡聚化合物的方法，鉴定优化的双链寡聚化合物的方法，选择优化的单链寡聚化合物的方法，方法 选择优化的双链寡聚化合物，鉴定多功能寡聚化合物的方法，用于优化用于调节RNA表达的靶区选择的方法，优化RNA表达调节的方法等。 本发明进一步提供低聚化合物，长度靶向靶RNA的8-80个核碱基，其中所述寡聚化合物与所述靶RNA杂交并且以单链和双链形式抑制至少50％的RNA水平，以及 多官能寡聚化合物。