Antisense oligonucleotides for inducing exon skipping and methods of use thereof
AssigneeThe University of Western Australia
InventorsStephen Donald WILTON, Sue FLETCHER, Graham MCCLOREY
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Abstract
Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described.
Claims3
(canceled)
An isolated antisense oligonucleotide 20 to 50 nucleotides comprising at least 17 bases of the sequence CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO:192), or an equivalent oligonucleotide, wherein said oligonucleotide, or its equivalent, induces exon 53 skipping in the human dystrophin pre-mRNA, and wherein the oligonucleotide comprises a modification, said modification comprising a base substitution of U by T.
An isolated antisense oligonucleotide of 24-50 nucleotides in length, said oligonucleotide comprising a sequence which is complementary to a target nucleic acid sequence of the human exon 53 pre-mRNA, wherein said target nucleic acid sequence comprises a 24 nucleotide sequence that is complementary to the sequence CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO:192), and wherein the oligonucleotide comprises a modification, said modification comprising a base substitution of U by T.
Dependencies are inferred from the claim text.