Antisense oligonucleotides for inducing exon skipping and methods of use thereof
First Claim
Patent Images
1. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 20 consecutive nucleotides of SEQ ID NO:
- 193, wherein the oligonucleotide specifically hybridizes to an exon 53 target region of the human dystrophin gene inducing exon 53 skipping, and wherein the uracil bases are optionally thymine bases.
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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.
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Citations
64 Claims
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1. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 20 consecutive nucleotides of SEQ ID NO:
- 193, wherein the oligonucleotide specifically hybridizes to an exon 53 target region of the human dystrophin gene inducing exon 53 skipping, and wherein the uracil bases are optionally thymine bases.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 36, 37, 38, 39, 40, 42, 43)
- 19. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 20 consecutive nucleotides complementary to an exon 53 target region of the human dystrophin gene designated as annealing site H53A(+39+69), wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 53 skipping, and wherein uracil bases in the antisense oligonucleotide are optionally thymine bases.
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44. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and a pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for intravenous administration. - View Dependent Claims (45)
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46. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and phosphate-buffered saline; wherein the injectable solution is formulated for intravenous administration.
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47. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and a pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for parenteral administration. - View Dependent Claims (48)
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49. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and phosphate-buffered saline; wherein the injectable solution is formulated for parenteral administration.
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50. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and a pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for intramuscular administration. - View Dependent Claims (51)
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52. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and phosphate-buffered saline; wherein the injectable solution is formulated for intramuscular administration.
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53. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and a pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for subcutaneous administration. - View Dependent Claims (54)
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55. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and phosphate-buffered saline; wherein the injectable solution is formulated for subcutaneous administration.
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56. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and a pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for intraocular administration. - View Dependent Claims (57)
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58. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and phosphate-buffered saline; wherein the injectable solution is formulated for intraocular administration.
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59. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and a pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for oral administration. - View Dependent Claims (60)
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61. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and phosphate-buffered saline; wherein the injectable solution is formulated for oral administration.
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62. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and a pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for transdermal administration. - View Dependent Claims (63)
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64. An injectable solution comprising:
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a morpholino antisense oligonucleotide 25 bases in length comprising a base sequence that is 100% complementary to 25 consecutive nucleotides of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO;
193), in which the uracil bases are thymine bases, andwherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and phosphate-buffered saline; wherein the injectable solution is formulated for transdermal administration.
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Specification