MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE
First Claim
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1. An isolated antisense oligonucleotide of 15 to 80 nucleotides comprising at least 15 bases of the sequence cuguugccuccgguucug (SEQ ID NO:
- 29), wherein said oligonucleotide induces exon 53 skipping in the human dystrophin pre-mRNA, wherein each internucleoside linkage of the oligonucleotide is a phosphorothioate linkage.
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Abstract
The invention relates to oligonucleotides for inducing skipping of exon 53 of the dystrophin gene. The invention also relates to methods of inducing exon 53 skipping using the oligonucleotides.
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Citations
23 Claims
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1. An isolated antisense oligonucleotide of 15 to 80 nucleotides comprising at least 15 bases of the sequence cuguugccuccgguucug (SEQ ID NO:
- 29), wherein said oligonucleotide induces exon 53 skipping in the human dystrophin pre-mRNA, wherein each internucleoside linkage of the oligonucleotide is a phosphorothioate linkage.
- View Dependent Claims (5, 6, 11, 15, 19, 20, 21, 23)
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2. An isolated antisense oligonucleotide of 15 to 80 nucleotides comprising at least 15 bases of the sequence cuguugccuccgguucug (SEQ ID NO:
- 29), wherein said oligonucleotide induces exon 53 skipping in the human dystrophin pre-mRNA, wherein each internucleoside linkage of the oligonucleotide is a phosphorodiamidate internucleoside linkage.
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3. An isolated antisense oligonucleotide of 18 to 80 nucleotides comprising at least the base sequence cuguugccuccgguucug (SEQ ID NO:
- 29), wherein said oligonucleotide induces exon 53 skipping in the human dystrophin pre-mRNA, wherein each internucleoside linkage of the oligonucleotide is a phosphorothioate linkage.
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4. An isolated antisense oligonucleotide of 18 to 80 nucleotides comprising at least the base sequence cuguugccuccgguucug (SEQ ID NO:
- 29), wherein said oligonucleotide induces exon 53 skipping in the human dystrophin pre-mRNA, wherein each internucleoside linkage of the oligonucleotide is a phosphorodiamidate internucleoside linkage.
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7. An isolated antisense oligonucleotide of 18 to 50 nucleotides in length, wherein said oligonucleotide binds to an exon-internal sequence of exon 53 of the human dystrophin pre-mRNA and induces skipping of exon 53, and wherein h53AON1 (cuguugccuccgguucug) (SEQ ID NO:
- 29) binds to said exon-internal sequence of exon 53 pre-mRNA, wherein each internucleoside linkage of the oligonucleotide is a phosphorothioate linkage.
- View Dependent Claims (9, 10)
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8. An isolated antisense oligonucleotide of 18 to 50 nucleotides in length, wherein said oligonucleotide binds to an exon-internal sequence of exon 53 of the human dystrophin pre-mRNA and induces skipping of exon 53, and wherein h53AON1 (cuguugccuccgguucug) (SEQ ID NO:
- 29) binds to said exon-internal sequence of exon 53 pre-mRNA, wherein each internucleoside linkage of the oligonucleotide is a phosphorodiamidate internucleoside linkage.
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12. An isolated antisense oligonucleotide of 18 to 50 nucleotides in length, wherein said oligonucleotide is complementary to a consecutive part of between 16 and 50 nucleotides of an exon-internal sequence of exon 53 of the human dystrophin pre-mRNA and induces skipping of exon 53, and wherein h53AON1 (cuguugccuccgguucug) (SEQ ID NO:
- 29) binds to said exon-internal sequence of exon 53 pre-mRNA, wherein each internucleoside linkage of the oligonucleotide is a phosphorothioate linkage.
- View Dependent Claims (14, 16, 17, 18, 22)
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13. An isolated antisense oligonucleotide of 18 to 50 nucleotides in length, wherein said oligonucleotide is complementary to a consecutive part of between 16 and 50 nucleotides of an exon-internal sequence of exon 53 of the human dystrophin pre-mRNA and induces skipping of exon 53, and wherein h53AON1 (cuguugccuccgguucug) (SEQ ID NO:
- 29) binds to said exon-internal sequence of exon 53 pre-mRNA, wherein each internucleoside linkage of the oligonucleotide is a phosphorodiamidate internucleoside linkage.
Specification