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 17 consecutive nucleotides of SEQ ID NO:
- 207, wherein the oligonucleotide specifically hybridizes to an exon 45 acceptor splice site of a human dystrophin gene, inducing exon 45 skipping, and wherein the uracil bases 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.
137 Citations
69 Claims
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1. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 17 consecutive nucleotides of SEQ ID NO:
- 207, wherein the oligonucleotide specifically hybridizes to an exon 45 acceptor splice site of a human dystrophin gene, inducing exon 45 skipping, and wherein the uracil bases are optionally thymine bases.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 65, 68, 69)
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23. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 17 consecutive nucleotides complementary to an exon 45 target region of a human dystrophin gene designated as annealing site H45A(−
- 06+20), wherein the antisense oligonucleotide specifically hybridizes to the acceptor splice site inducing exon 45 skipping, and wherein uracil bases in the antisense oligonucleotide are optionally thymine bases.
- View Dependent Claims (24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36, 37, 38, 39, 40, 41, 42, 43, 44, 45, 46, 66)
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47. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 20 consecutive nucleotides of SEQ ID NO:
- 207, wherein the uracil bases are optionally thymine bases.
- View Dependent Claims (48, 49, 50, 51, 52, 53, 54, 55, 56, 57, 58, 59, 60, 61, 62, 63, 64, 67)
Specification