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 20 consecutive nucleotides of SEQ ID NO:
- 167, wherein the oligonucleotide specifically hybridizes to an exon 44 acceptor splice site of the human dystophin gene inducing exon 44 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.
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Citations
43 Claims
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1. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 20 consecutive nucleotides of SEQ ID NO:
- 167, wherein the oligonucleotide specifically hybridizes to an exon 44 acceptor splice site of the human dystophin gene inducing exon 44 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, 36, 37, 38, 40, 41, 42, 43)
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19. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 20 consecutive nucleotides complementary to an exon 44 target region of the human dystophin gene designated as annealing site H44A(−
- 06+14), wherein the antisense oligonucleotide specifically hybridizes to the acceptor splice site inducing exon 44 skipping, and wherein uracil bases in the antisense oligonucleotide are optionally thymine bases.
- View Dependent Claims (20, 21, 22, 23, 24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 39)
Specification