ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF
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Abstract
Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described.
42 Citations
49 Claims
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1-20. -20. (canceled)
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21. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 20 nucleotides of SEQ ID NO:
- 193, wherein at least one pyrimidine base of the oligonucleotide comprises a 5-substituted pyrimidine base and uracil bases are optionally thymine bases, and wherein the oligonucleotide specifically hybridizes to an exon 53 target region of the human dystrophin gene inducing exon 53 skipping.
- View Dependent Claims (22, 23, 24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36, 48)
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37. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 20 nucleotides of SEQ ID NO:
- 193, wherein at least one purine base of the oligonucleotide comprises an N-2, N-6 substituted purine base and uracil bases are optionally thymine bases, and wherein the oligonucleotide specifically hybridizes to an exon 53 target region of the human dystrophin gene inducing exon 53 skipping.
- View Dependent Claims (38, 39, 40, 41, 42, 43, 44, 45, 46, 47, 49)
Specification