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.
38 Citations
28 Claims
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1-20. -20. (canceled)
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21. An antisense oligonucleotide of 24 bases comprising SEQ ID NO:
- 1, in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′
-O-methyl phosphorothioate oligoribonucleotide;
or a pharmaceutically acceptable salt thereof. - View Dependent Claims (23)
- 1, in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′
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22. An antisense oligonucleotide of 24 bases consisting of SEQ ID NO:
- 1, wherein the antisense oligonucleotide is a 2′
-O-methyl phosphorothioate oligoribonucleotide;
or a pharmaceutically acceptable salt thereof. - View Dependent Claims (24)
- 1, wherein the antisense oligonucleotide is a 2′
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25. An antisense oligonucleotide of 21 bases comprising SEQ ID NO:
- 2, in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′
-O-methyl phosphorothioate oligoribonucleotide;
or a pharmaceutically acceptable salt thereof. - View Dependent Claims (27)
- 2, in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′
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26. An antisense oligonucleotide of 21 bases consisting of SEQ ID NO:
- 2, wherein the antisense oligonucleotide is a 2′
-O-methyl phosphorothioate oligoribonucleotide;
or a pharmaceutically acceptable salt thereof. - View Dependent Claims (28)
- 2, wherein the antisense oligonucleotide is a 2′
Specification