Antisense oligonucleotides for inducing exon skipping and methods of use thereof
First Claim
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1. 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.
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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.
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Citations
8 Claims
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1. 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 (3)
- 1, in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′
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2. 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 (4)
- 1, wherein the antisense oligonucleotide is a 2′
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5. An antisense oligonucleotide of 1 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 (7)
- 2, in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′
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6. An antisense oligonucleotide of 1 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 (8)
- 2, wherein the antisense oligonucleotide is a 2′
Specification