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.
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Citations
44 Claims
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1-14. -14. (canceled)
- 15. isolated antisense oligonucleotide comprising UGCCAUCCUG (base positions 5-14 of SEQ ID NO. 207), wherein the oligonucleotide specifically hybridizes to an exon 45 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
- 24. A method of inducing exon-skipping of dystrophin exon 45, 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 UGCCAUCCUG (base positions 5-14 of SEQ ID NO. 207), wherein the oligonucleotide specifically hybridizes to an exon 45 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
- 33. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising UGCCAUCCUG (base positions 5-14 of SEQ ID NO. 207), wherein the uracil bases are optionally thymidine bases.
- 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 UGCCAUCCUG (base positions 5-14 of SEQ ID NO. 207), wherein the oligonucleotide specifically hybridizes to an exon 45 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 45, and wherein the uracil bases are optionally thymidine bases.
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41. An isolated antisense oligonucleotide comprising CCAUCCUGGA (base positions 7-16 of SEQ ID NO. 207), wherein the oligonucleotide specifically hybridizes to an exon 45 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
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42. A method of inducing exon-skipping of dystrophin exon 45, 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 CCAUCCUGGA (base positions 7-16 of SEQ ID NO. 207), wherein the oligonucleotide specifically hybridizes to an exon 45 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
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43. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising CCAUCCUGGA (base positions 7-16 of SEQ ID NO. 207), wherein the uracil bases are optionally thymidine bases.
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44. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising CCAUCCUGGA (base positions 7-16 of SEQ ID NO. 207), wherein the oligonucleotide specifically hybridizes to an exon 45 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 45, and wherein the uracil bases are optionally thymidine bases.
Specification