Chimeric oligomeric compounds for modulation of splicing
AssigneesIsis Pharmaceuticals, Inc., Sarepta Therapeutics, Inc.
InventorsC. Frank Bennett, Nicholas M. Dean, Ryszard M. Kole, Casey C. Kopczynski
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Abstract
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.
Claims20
-57. (canceled)
A chimeric RNase H-independent antisense compound 13 to 80 nucleobases in length targeted to a selected target mRNA, wherein the compound comprises:
- a modified sugar moiety at the 5′-most nucleoside;
- a modified sugar moiety at the 3′-most nucleoside;
- internal nucleosides comprising 2, 3, 4 or 5 separate regions of 2′-deoxyribonucleosides, wherein each region consists of 1, 2, 3 or 4 nucleosides;
- and
- at least one nucleoside with a modified sugar moiety separating each region of 2′-deoxynucleosides.
Dependencies are inferred from the claim text.