CPC C12N 15/113 (2013.01) [A61K 31/4166 (2013.01); A61K 31/712 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/3233 (2013.01); C12N 2310/33 (2013.01)] | 22 Claims |
1. An anti-sense compound comprising a modified splice-switching oligonucleotide (SSO) consisting of 22 to 30 linked nucleosides and having a nucleobase sequence at least 80% complementary to the corresponding nucleotide sequence within SEQ ID NO:5, wherein the SSO comprises a sequence selected from the group consisting of SEQ ID NO:7, SEQ ID NO:9, SEQ ID NO: 11 and SEQ ID NO: 13, and a sequence with at least 80% identity to SEQ ID NO:7, SEQ ID NO:9, SEQ ID NO: 11 or SEQ ID NO: 13, wherein each nucleoside of the SSO comprises a sugar modification.
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