ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF
First Claim
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1. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 10 consecutive nucleotides of a nucleotide sequence selected from the group consisting of SEQ ID NOs:
- 1-212, wherein the oligonucleotide specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping, and wherein uracil bases in the antisense oligonucleotide are optionally thymine bases.
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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.
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Citations
20 Claims
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1. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 10 consecutive nucleotides of a nucleotide sequence selected from the group consisting of SEQ ID NOs:
- 1-212, wherein the oligonucleotide specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping, and wherein uracil bases in the antisense oligonucleotide are optionally thymine bases.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 16, 17, 18, 19, 20)
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15. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 10 consecutive nucleotides complementary to a target region of an exon in the human dystrophin gene designated as an annealing site selected from the group set forth in Table 1A, wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon skipping.
Specification