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
46 Claims
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1-14. -14. (canceled)
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15. An isolated antisense oligonucleotide comprising CUCUUUUCCA (base positions 1-10 of SEQ ID NO:
- 203), wherein the oligonucleotide specifically hybridizes to an exon 46 target region of a Dystrophin gene and has ability to skip exon 46, 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 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 comprising CUCUUUUCCA (base positions 1-10 of SEQ ID NO:
- 203), wherein the oligonucleotide specifically hybridizes to an exon 46 target region of a Dystrophin gene and has ability to skip exon 46, 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. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising CUCUUUUCCA (base positions 1-10 of SEQ ID NO:
- 203), wherein the uracil bases are optionally thymidine bases.
- View Dependent Claims (36, 37, 38)
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39. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising CUCUUUUCCA (base positions 1-10 of SEQ ID NO:
- 203), wherein the oligonucleotide specifically hybridizes to an exon 46 target region of a Dystrophin gene and has ability to skip exon 46, wherein said uracil bases are optionally thymidine bases.
- View Dependent Claims (40, 41, 42)
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43. An isolated antisense oligonucleotide comprising GCUCUUUUCC (base positions 22-31 of SEQ ID NO:
- 204), wherein the oligonucleotide specifically hybridizes to an exon 46 target region of a Dystrophin gene and has ability to skip exon 46, wherein said uracil bases are optionally thymidine bases.
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44. 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 comprising GCUCUUUUCC (base positions 22-31 of SEQ ID NO:
- 204), wherein the oligonucleotide specifically hybridizes to an exon 46 target region of a Dystrophin gene and has ability to skip exon 46, wherein said uracil bases are optionally thymidine bases.
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45. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising GCUCUUUUCC (base positions 22-31 of SEQ ID NO:
- 204), wherein the uracil bases are optionally thymidine bases.
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46. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising GCUCUUUUCC (base positions 22-31 of SEQ ID NO:
- 204), wherein the oligonucleotide specifically hybridizes to an exon 46 target region of a Dystrophin gene and has ability to skip exon 46, wherein said uracil bases are optionally thymidine bases.
Specification