Oligonucleotide comprising an inosine for treating DMD
First Claim
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1. An isolated antisense oligonucleotide consisting of the base sequence of SEQ ID NO:
- 557, provided that said isolated antisense oligonucleotide comprises at least one position wherein a guanosine base is substituted with an inosine base, wherein said isolated antisense oligonucleotide comprises a modification and induces skipping of said exon 45 of human dystrophin pre-mRNA.
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Abstract
The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.
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12 Claims
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1. An isolated antisense oligonucleotide consisting of the base sequence of SEQ ID NO:
- 557, provided that said isolated antisense oligonucleotide comprises at least one position wherein a guanosine base is substituted with an inosine base, wherein said isolated antisense oligonucleotide comprises a modification and induces skipping of said exon 45 of human dystrophin pre-mRNA.
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