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 18 nucleotides consisting of the base sequence of the sequence cuguugccuccgguucug (SEQ ID NO:
- 29), wherein said oligonucleotide induces exon 53 skipping in the human dystrophin pre-mRNA, said oligonucleotide comprising a modification selected from the group consisting of;
2′
-O- methyl, 2′
-O-methylphosphorothioate, a morpholine ring, a phosphorodiamidate linkage, a peptide nucleic acid and a locked nucleic acid.
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Abstract
The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.
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1 Claim
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1. An isolated antisense oligonucleotide of 18 nucleotides consisting of the base sequence of the sequence cuguugccuccgguucug (SEQ ID NO:
- 29), wherein said oligonucleotide induces exon 53 skipping in the human dystrophin pre-mRNA, said oligonucleotide comprising a modification selected from the group consisting of;
2′
-O- methyl, 2′
-O-methylphosphorothioate, a morpholine ring, a phosphorodiamidate linkage, a peptide nucleic acid and a locked nucleic acid.
- 29), wherein said oligonucleotide induces exon 53 skipping in the human dystrophin pre-mRNA, said oligonucleotide comprising a modification selected from the group consisting of;
Specification