Antisense Oligonucleotides for Inducing Exon Skipping and Methods of Use Thereof
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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.
71 Citations
44 Claims
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1-14. -14. (canceled)
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15. An isolated antisense oligonucleotide comprising AACUGUUGCC (base positions 6-15 of SEQ ID NO:
- 193), wherein the oligonucleotide specifically hybridizes to an exon 53 target region of the Dystrophin, wherein said uracil bases are optionally thymidine bases.
- View Dependent Claims (16, 17, 18, 19, 20, 21, 22, 23, 24)
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25. A method of inducing exon-skipping of dystrophin exon 53, 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 comprising AACUGUUGCC (base positions 6-15 of SEQ ID NO:
- 193), wherein the oligonucleotide specifically hybridizes to an exon 53 target region of the Dystrophin gene, wherein said uracil bases are optionally thymidine bases.
- View Dependent Claims (26, 27, 28, 29, 30, 31, 32, 33, 34)
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35. An isolated antisense oligonucleotide comprising CCUUGGUUUC (base positions 7-16 of SEQ ID NO:
- 194) wherein the oligonucleotide specifically hybridizes to an exon 53 target region of the Dystrophin, wherein said uracil bases are optionally thymidine bases.
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36. A method of inducing exon-skipping of dystrophin exon 53, 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 comprising CCUUGGUUUC (base positions 7-16 of SEQ ID NO:
- 194) wherein the oligonucleotide specifically hybridizes to an exon 53 target region of the Dystrophin gene, wherein said uracil bases are optionally thymidine bases.
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37. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising AACUGUUGCC (base positions 6-15 of SEQ ID NO:
- 193), wherein the uracil bases are optionally thymidine bases.
- View Dependent Claims (38, 39, 40)
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41. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising AACUGUUGCC (base positions 6-15 of SEQ ID NO:
- 193), wherein the oligonucleotide specifically hybridizes to an exon 53 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 53, and wherein the uracil bases are optionally thymidine bases.
- View Dependent Claims (42, 43, 44)
Specification