发明名称 |
Chimeric oligomeric compounds for modulation of splicing |
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.
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申请公布号 |
US8501703(B2) |
申请公布日期 |
2013.08.06 |
申请号 |
US20060065250 |
申请日期 |
2006.08.29 |
申请人 |
BENNETT C. FRANK;DEAN NICHOLAS M.;KOLE RYSZARD;KOPCZYNSKI CASEY C.;ISIS PHARMACEUTICALS, INC.;SAREPTA THERAPEUTICS |
发明人 |
BENNETT C. FRANK;DEAN NICHOLAS M.;KOLE RYSZARD;KOPCZYNSKI CASEY C. |
分类号 |
A61K48/00 |
主分类号 |
A61K48/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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