ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF
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Accused Products
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
75 Claims
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1-14. -14. (canceled)
- 15. An isolated antisense oligonucleotide of about 10 to about 50 nucleotides in length comprising a sequence that is specifically hybridizable to an exon 46 target region of the Dystrophin gene designated as annealing site H46D(+16-04), or a combination thereof.
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38. An isolated antisense oligonucleotide wherein the antisense oligonucleotide consists of the sequence identified as SEQ ID NO:
- 168, SEQ ID NO;
169, SEQ ID NO;
203, SEQ ID NO;
204, SEQ ID NO;
205 or SEQ ID NO;
206, optionally containing a nucleobase substitution, wherein the uracil bases are optionally thymine bases. - View Dependent Claims (39)
- 168, SEQ ID NO;
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40. An isolated antisense oligonucleotide of about 10 to about 50 nucleotides in length wherein the antisense oligonucleotide is specifically hybridizable to annealing site H46A(+86+115), H46A(+107+137) or a combination thereof.
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41. An isolated antisense oligonucleotide of about 10 to about 50 nucleotides in length comprising a sequence wherein the antisense oligonucleotide is 100% complementary to annealing site H46A(+86+115), H46A(+107+137) or a combination thereof, or contains a nucleobase substitution.
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42. An isolated antisense oligonucleotide of about 10 to about 50 nucleotides in length comprising a sequence, wherein the antisense oligonucleotide is 100% complementary to annealing site H46D(+16-04), H46A(+90+109), H46A(+86+115), H46A(+107+137), H46A(−
- 10+20), H46A(+50+77) or a combination thereof, or contains a nucleobase substitution.
- 51. An isolated antisense oligonucleotide of at least 10 nucleotides in length that specifically hybridizes to an exon 46 target region of a Dystrophin gene wherein the oligonucleotide comprises annealing site H46A (+106+115) and has ability to skip exon 46.
- 58. A method of inducing exon-skipping of dystrophin exon 46, comprising introducing a nucleic acid molecule into a cell by way of an expression vector, wherein the nucleic acid molecule is an isolated antisense oligonucleotide of at least 10 nucleotides in length that specifically hybridizes to an exon 46 target region of a Dystrophin gene wherein the oligonucleotide comprises annealing site H46A (+106+115) and has ability to skip exon.
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68. An isolated antisense oligonucleotide of at least 10 nucleotides in length that specifically hybridizes to an exon 46 target region of a Dystrophin gene wherein the oligonucleotide comprises annealing site H46A(+107+116) and has ability to skip exon 46.
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69. A method of inducing exon-skipping of dystrophin exon 46, comprising introducing a nucleic acid molecule into a cell by way of an expression vector, wherein the nucleic acid molecule is an isolated antisense oligonucleotide of at least 10 nucleotides in length that specifically hybridizes to an exon 46 target region of a Dystrophin gene wherein the oligonucleotide comprises annealing site H46A(+107+116) and has ability to skip exon 46.
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70. An isolated antisense oligonucleotide of 17 to 30 nucleotides in length comprising a sequence that is specifically hybridizable to an exon 46 target region of the Dystrophin gene designated as annealing site H46D(+16-04), H46A(−
- 10+20), or a combination thereof.
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71. An isolated antisense oligonucleotide of 17 to 30 nucleotides in length wherein the antisense oligonucleotide is specifically hybridizable to annealing site H46A(+86+115), H46A(+107+137) or a combination thereof.
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72. An isolated antisense oligonucleotide of 17 to 30 nucleotides in length comprising a sequence wherein the antisense oligonucleotide is 100% complementary to annealing site H46A(+86+115), H46A(+107+137) or a combination thereof, or contains a nucleobase substitution.
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73. An isolated antisense oligonucleotide of 17 to 30 nucleotides in length comprising a sequence, wherein the antisense oligonucleotide is 100% complementary to annealing site H46D(+16-04), H46A(+90+109), H46A(+86+115), H46A(+107+137), H46A(−
- 10+20), H46A(+50+77) or a combination thereof, or contains a nucleobase substitution.
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74. An isolated antisense oligonucleotide of 17 nucleotides in length that specifically hybridizes to an exon 46 target region of a Dystrophin gene wherein the oligonucleotide comprises annealing site H46A (+106+115) and has ability to skip exon 46.
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75. An isolated antisense oligonucleotide of 17 nucleotides in length that specifically hybridizes to an exon 46 target region of a Dystrophin gene wherein the oligonucleotide comprises annealing site H46A(+107+116) and has ability to skip exon 46.
Specification