Antisense Oligonucleotides for Inducing Exon Skipping and Methods of Use Thereof
2 Assignments
0 Petitions
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.
81 Citations
56 Claims
-
1-14. -14. (canceled)
- 15. An isolated antisense oligonucleotide comprising at least 10 nucleotides of SEQ ID NO. 167 wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene.
- 24. A method of inducing exon-skipping of dystrophin exon 44, 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 at least 10 nucleotides of SEQ ID NO. 167 wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene.
- 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 at least 10 nucleotides of SEQ ID NO. 167.
- 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 at least 10 nucleotides of SEQ ID NO. 167, wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 44, and wherein the uracil bases are optionally thymidine bases.
-
41. An isolated antisense oligonucleotide comprising UCAAAUCGCC (base positions 6-15 of SEQ ID NO:
- 167), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
-
42. A method of inducing exon-skipping of dystrophin exon 44, 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 an isolated antisense oligonucleotide comprising UCAAAUCGCC (base positions 6-15 of SEQ ID NO:
- 167), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
-
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 UCAAAUCGCC (base positions 6-15 of SEQ ID NO:
- 167), wherein the uracil bases are optionally thymidine bases.
-
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 UCAAAUCGCC (base positions 6-15 of SEQ ID NO:
- 167), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 44, and wherein the uracil bases are optionally thymidine bases.
-
45. An isolated antisense oligonucleotide comprising UCGCCUGCAG (base positions 11-20 of SEQ ID NO:
- 167), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
-
46. A method of inducing exon-skipping of dystrophin exon 44, 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 an isolated antisense oligonucleotide comprising UCGCCUGCAG (base positions 11-20 of SEQ ID NO:
- 167), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
-
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 UCGCCUGCAG (base positions 11-20 of SEQ ID NO:
- 167), wherein the uracil bases are optionally thymidine bases.
-
48. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising UCGCCUGCAG (base positions 11-20 of SEQ ID NO:
- 167), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 44, and wherein the uracil bases are optionally thymidine bases.
-
49. An isolated antisense oligonucleotide comprising AUCUGUCAAA (base positions 1-10 of SEQ ID NO:
- 167), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
-
50. A method of inducing exon-skipping of dystrophin exon 44, 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 an isolated antisense oligonucleotide comprising AUCUGUCAAA (base positions 1-10 of SEQ ID NO:
- 167), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
-
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 AUCUGUCAAA (base positions 1-10 of SEQ ID NO:
- 167), wherein the uracil bases are optionally thymidine bases.
-
52. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising AUCUGUCAAA (base positions 1-10 of SEQ ID NO:
- 167), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 44, and wherein the uracil bases are optionally thymidine bases.
-
53. An isolated antisense oligonucleotide comprising CUUUCUGAGA (base positions 8-17 of SEQ ID NO:
- 165), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
-
54. A method of inducing exon-skipping of dystrophin exon 44, 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 an isolated antisense oligonucleotide comprising CUUUCUGAGA (base positions 8-17 of SEQ ID NO:
- 165), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene, wherein the uracil bases are optionally thymidine bases.
-
55. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising CUUUCUGAGA (base positions 8-17 of SEQ ID NO:
- 165), wherein the uracil bases are optionally thymidine bases.
-
56. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an isolated antisense oligonucleotide comprising CUUUCUGAGA (base positions 8-17 of SEQ ID NO:
- 165), wherein the oligonucleotide specifically hybridizes to an exon 44 target region of a Dystrophin gene and induces exon-skipping of dystrophin exon 44, and wherein the uracil bases are optionally thymidine bases.
Specification