ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF
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Abstract
Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described.
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Citations
3 Claims
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1. (canceled)
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2. An isolated antisense oligonucleotide 20 to 50 nucleotides comprising at least 17 bases of the sequence CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO:
- 192), or an equivalent oligonucleotide, wherein said oligonucleotide, or its equivalent, induces exon 53 skipping in the human dystrophin pre-mRNA, and wherein the oligonucleotide comprises a modification, said modification comprising a base substitution of U by T.
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3. An isolated antisense oligonucleotide of 24-50 nucleotides in length, said oligonucleotide comprising a sequence which is complementary to a target nucleic acid sequence of the human exon 53 pre-mRNA, wherein said target nucleic acid sequence comprises a 24 nucleotide sequence that is complementary to the sequence CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO:
- 192), and wherein the oligonucleotide comprises a modification, said modification comprising a base substitution of U by T.
Specification