Patent ID: 11866689
Assignee: DUKE UNIVERSITY
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
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.