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
EP1937312(B1)
申请公布日期
2016.06.29
申请号
EP20060802842
申请日期
2006.08.29
申请人
IONIS PHARMACEUTICALS, INC.;ERCOLE BIOTECH, INC.
发明人
BENNETT, C. FRANK;DEAN, NICHOLAS, M.;KOLE, RYSZARD;KOPCZYNSKI, CASEY, C.