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.
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
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