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
An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.
Claims7
(canceled)
An isolated antisense oligonucleotide of 20 to 50 nucleotides in length, said oligonucleotide comprising a nucleotide sequence which is complementary to a target sequence of exon 51 of the human dystrophin pre-mRNA, wherein said target sequence comprises a nucleotide sequence that is complementary to the sequence ACA UCA AGG AAG AUG GCA UUU CUA G (SEQ ID NO:
- 180), and wherein said oligonucleotide comprises a morpholine ring.
An isolated antisense oligonucleotide of 20 to 50 nucleotides in length, said oligonucleotide comprising a nucleotide sequence which is complementary to a target sequence of exon 51 of the human dystrophin pre-mRNA, wherein said target sequence comprises a nucleotide sequence that is complementary to the sequence ACA UCA AGG AAG AUG GCA UUU CUA G (SEQ ID NO:
- 180), and wherein said oligonucleotide comprises a peptide nucleic acid and/or locked nucleic acid.
An isolated antisense oligonucleotide of 20 to 50 nucleotides in length, said oligonucleotide comprising a nucleotide sequence which is complementary to a target sequence of exon 51 of the human dystrophin pre-mRNA, wherein said target sequence comprises a nucleotide sequence that is complementary to the sequence ACA UCA AGG AAG AUG GCA UUU CUA G (SEQ ID NO:
- 180), and wherein said oligonucleotide comprises a 2′-O-methyl ribose moiety.
Dependencies are inferred from the claim text.