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
51 Claims
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1-14. -14. (canceled)
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15. An isolated antisense oligonucleotide comprising GCAUUUCUAG (base positions 16-25 of SEQ ID NO:
- 180), wherein the oligonucleotide specifically hybridizes to an exon 51 target region of a Dystrophin gene and wherein the oligonucleotide has ability to skip exon 51, wherein the uracil bases are optionally thymine bases.
- View Dependent Claims (16, 17, 18, 19, 20, 21, 22, 23)
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24. A method of inducing exon-skipping of dystrophin exon 51, 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 GCAUUUCUAG (base positions 16-25 of SEQ ID NO:
- 180), wherein the oligonucleotide specifically hybridizes to an exon 51 target region of a Dystrophin gene and wherein the oligonucleotide has ability to skip exon 51, wherein the uracil bases are optionally thymine bases.
- View Dependent Claims (25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35)
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36. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising GCAUUUCUAG (base positions 16-25 of SEQ ID NO:
- 180), wherein the uracil bases are optionally thymidine bases.
- View Dependent Claims (37, 38, 39)
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40. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising GCAUUUCUAG (base positions 16-25 of SEQ ID NO:
- 180), wherein the oligonucleotide specifically hybridizes to an exon 51 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 51, and wherein the uracil bases are optionally thymidine bases.
- View Dependent Claims (41, 42, 43)
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44. An isolated antisense oligonucleotide comprising UCCAACAUCA (base positions 2-11 of SEQ ID NO:
- 181), wherein the oligonucleotide specifically hybridizes to an exon 51 target region of a Dystrophin gene and wherein the oligonucleotide has ability to skip exon 51, wherein the uracil bases are optionally thymine bases.
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45. A method of inducing exon-skipping of dystrophin exon 51, 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 UCCAACAUCA (base positions 2-11 of SEQ ID NO:
- 181), wherein the oligonucleotide specifically hybridizes to an exon 51 target region of a Dystrophin gene and wherein the oligonucleotide has ability to skip exon 51, wherein the uracil bases are optionally thymine 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 UCCAACAUCA (base positions 2-11 of SEQ ID NO:
- 181), wherein the uracil bases are optionally thymidine bases.
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47. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising UCCAACAUCA (base positions 2-11 of SEQ ID NO:
- 181), wherein the oligonucleotide specifically hybridizes to an exon 51 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 51, and wherein the uracil bases are optionally thymidine bases.
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48. An isolated antisense oligonucleotide comprising CUCCAACAUCAAGGAAG (based positions 1-17 of SEQ ID NO:
- 181), wherein the oligonucleotide specifically hybridizes to an exon 51 target region of a Dystrophin gene and wherein the oligonucleotide has ability to skip exon 51, wherein the uracil bases are optionally thymine bases.
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49. A method of inducing exon-skipping of dystrophin exon 51, 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 CUCCAACAUCAAGGAAG (based positions 1-17 of SEQ ID NO:
- 181), wherein the oligonucleotide specifically hybridizes to an exon 51 target region of a Dystrophin gene and wherein the oligonucleotide has ability to skip exon 51, wherein the uracil bases are optionally thymine bases.
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50. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising CUCCAACAUCAAGGAAG (based positions 1-17 of SEQ ID NO:
- 181), wherein the uracil bases are optionally thymidine bases.
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51. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising CUCCAACAUCAAGGAAG (based positions 1-17 of SEQ ID NO:
- 181), wherein the oligonucleotide specifically hybridizes to an exon 51 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 51, and wherein the uracil bases are optionally thymidine bases.
Specification