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US 11,142,767 B2Granted
Antisense oligonucleotides that bind to exon 51 of human dystrophin pre-mRNA
AssigneeThe Governors of the University of Alberta
InventorsToshifumi Yokota, Yusuke Echigoya
Life of this patent
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Abstract
The present invention relates to a therapeutic antisense oligonucleotide which binds to exon 51 of the human dystrophin pre-mRNA to induce exon skipping, and conjugates and compositions thereof for the treatment of DMD.
Claims9
A morpholino antisense oligonucleotide (PMO) consisting of SEQ ID NO:1 (Ac0), SEQ ID NO:2 (Ac5), SEQ ID NO:3 (Ac26), SEQ ID NO:4 (Ac30), or SEQ ID NO:5 (Ac48), wherein the PMO binds to the human dystrophin pre-mRNA of exon 51 within the region between 0 and +89 of the pre-mRNA sequence and induces exon skipping of exon 51 of the human dystrophin pre-mRNA.
Dependencies are inferred from the claim text.