MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD
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Accused Products
Abstract
Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.
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Citations
73 Claims
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1-65. -65. (canceled)
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66. An antisense oligonucleotide of 24 bases comprising a base sequence that is 100% complementary to 24 consecutive bases of exon 55 of the human dystrophin pre-mRNA, wherein the base sequence comprises 20 consecutive bases of TCTATGAGTTTCTTCCAAAGCAGCC (SEQ ID NO:
- 527) in which thymine bases are uracil bases, wherein the antisense oligonucleotide is a 2′
-O-methyl oligonucleotide, and wherein the antisense oligonucleotide induces exon 55 skipping;
or a pharmaceutically acceptable salt thereof. - View Dependent Claims (70)
- 527) in which thymine bases are uracil bases, wherein the antisense oligonucleotide is a 2′
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67. An antisense oligonucleotide of 24 bases comprising a base sequence that is 100% complementary to 24 consecutive bases of exon 55 of the human dystrophin pre-mRNA, wherein the base sequence comprises 20 consecutive bases of TCTATGAGTTTCTTCCAAAGCAGCC (SEQ ID NO:
- 527) in which;
(i) thymine bases are uracil bases and (ii) cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′
-O-methyl oligonucleotide, and wherein the antisense oligonucleotide induces exon 55 skipping;
or a pharmaceutically acceptable salt thereof. - View Dependent Claims (71)
- 527) in which;
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68. An antisense oligonucleotide of 24 bases comprising a base sequence that is 100% complementary to 24 consecutive bases of exon 55 of the human dystrophin pre-mRNA, wherein the base sequence comprises 20 consecutive bases of TCTATGAGTTTCTTCCAAAGCAGCC (SEQ ID NO:
- 527) in which;
(i) thymine bases are uracil bases and (ii) one or more of the bases are hypoxanthine, wherein the antisense oligonucleotide is a 2′
-O-methyl oligonucleotide, and wherein the antisense oligonucleotide induces exon 55 skipping;
or a pharmaceutically acceptable salt thereof. - View Dependent Claims (72)
- 527) in which;
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69. An antisense oligonucleotide of 24 bases comprising a base sequence that is 100% complementary to 24 consecutive bases of exon 55 of the human dystrophin pre-mRNA, wherein the base sequence comprises 20 consecutive bases of TCTATGAGTTTCTTCCAAAGCAGCC (SEQ ID NO:
- 527) in which;
(i) thymine bases are uracil bases, (ii) one or more of the bases are hypoxanthine, and (iii) cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′
-O-methyl oligonucleotide, and wherein the antisense oligonucleotide induces exon 55 skipping;
or a pharmaceutically acceptable salt thereof. - View Dependent Claims (73)
- 527) in which;
Specification