Patent ID: 8501703
Filing Date: 2013-08-06
Classification: C12N

Abstract:
1. A method of modulating splicing of a selected target pre-mRNA in a cell comprising contacting the cell with an oligomeric compound comprising an oligonucleotide consisting of 13 to 30 linked nucleosides which is complementary to the selected target pre-mRNA, and wherein the oligonucleotide has 7-11 separate regions, wherein: the 5′-most region consists of 1-5 linked modified nucleosides, each comprising a modified sugar moiety; the 3′-most region consists of 1-5 linked modified nucleosides, each comprising a modified sugar moiety; and the internal regions alternate between regions of 1-5 linked 2′-deoxyribonucleosides and regions of 1-5 linked modified nucleosides, each comprising a modified sugar moiety, wherein splicing of the selected target pre-mRNA is modulated.