- 专利标题: Chimeric oligomeric compounds for modulation of splicing
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申请号: US12065250申请日: 2006-08-29
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公开(公告)号: US08501703B2公开(公告)日: 2013-08-06
- 发明人: C. Frank Bennett , Nicholas M. Dean , Ryszard Kole , Casey C. Kopczynski
- 申请人: C. Frank Bennett , Nicholas M. Dean , Ryszard Kole , Casey C. Kopczynski
- 申请人地址: US CA Carlsbad US MA Cambridge
- 专利权人: Isis Pharmaceuticals, Inc.,Sarepta Therapeutics
- 当前专利权人: Isis Pharmaceuticals, Inc.,Sarepta Therapeutics
- 当前专利权人地址: US CA Carlsbad US MA Cambridge
- 代理机构: Isis Pharmaceuticals, Inc. Patent Dept.
- 国际申请: PCT/US2006/034308 WO 20060829
- 国际公布: WO2007/028065 WO 20070308
- 主分类号: A61K48/00
- IPC分类号: A61K48/00
摘要:
Disclosed herein are compounds, compositions and methods for modulating splicing of a selected target mRNA. Further provided are uses of the disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Methods of enhancing cellular uptake, modulating tissue distribution and enhancing pharmacological activity of RNase H-independent antisense oligonucleotides are also provided.
公开/授权文献
- US20110039334A1 CHIMERIC OLIGOMERIC COMPOUNDS FOR MODULATION OF SPLICING 公开/授权日:2011-02-17
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