Patent ID: 9096851
Filing Date: 2015-08-04
Classification: A61P,C12N

Abstract:
1. A compound which comprises a modified oligonucleotide consisting of 12-30 linked nucleosides, at least a 12 nucleobase sequence portion of which is complementary to nucleotides within the region selected from nucleotides 718-751, 1388-1423, 1394-1423, 1659-1689, 2100-2129 and 1399-1423 of SEQ ID NO: 9 or 2728-2797 of SEQ ID NO:10 wherein the modified oligonucleotide comprises: (a) a gap segment consisting of linked deoxynucleosides; (b) a 5′ wing segment consisting of linked modified nucleosides; and (c) a 3′ wing segment consisting of linked modified nucleosides; wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment and wherein at least one modified nucleoside within each wing segment comprises a modified sugar.