Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
First Claim
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1. An isolated antisense oligonucleotide consisting of 7, 8, 9, or 10 nucleotides and comprising the base sequence of 5′
- -UCAGCUUCUGUUAGCCACUG-3′
(SEQ ID NO;
5), said oligonucleotide comprising a modification.
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Abstract
The invention relates to oligonucleotides that bind to and induce skipping of exon 44 in the human dystrophin pre-mRNA, and methods of use.
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Citations
15 Claims
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1. An isolated antisense oligonucleotide consisting of 7, 8, 9, or 10 nucleotides and comprising the base sequence of 5′
- -UCAGCUUCUGUUAGCCACUG-3′
(SEQ ID NO;
5), said oligonucleotide comprising a modification. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 12, 13, 14, 15)
- -UCAGCUUCUGUUAGCCACUG-3′
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10. An isolated antisense oligonucleotide comprising a phosphorothioate backbone consisting of 20 nucleotides having the base sequence of 5′
- -UCAGCUUCUGUUAGCCACUG-3′
(SEQ ID NO;
5), wherein each of the sugar moieties is 2′
-O-methyl substituted.
- -UCAGCUUCUGUUAGCCACUG-3′
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11. A viral-based vector comprising an expression cassette for expression of the oligonucleotide consisting of 7, 8, 9 or 10 nucleotides and comprising the base sequence of 5′
- -UCAGCUUCUGUUAGCCACUG-3′
(SEQ ID NO;
5).
- -UCAGCUUCUGUUAGCCACUG-3′
Specification