RNA Modulating Oligonucleotides with Improved Characteristics for the Treatment of Duchenne and Becker Muscular Dystrophy
First Claim
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1. An oligonucleotide comprising a 2′
- -O-methyl RNA monomer and a phosphorothioate backbone and comprising a 5-methyluracil and/or a 5-methylcytosine and/or a 2,6-diaminopurine base, said oligonucleotide being able to induce skipping of an exon of the dystrophin pre-mRNA.
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Abstract
The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing and/or delaying DMD or BMD.
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Citations
15 Claims
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1. An oligonucleotide comprising a 2′
- -O-methyl RNA monomer and a phosphorothioate backbone and comprising a 5-methyluracil and/or a 5-methylcytosine and/or a 2,6-diaminopurine base, said oligonucleotide being able to induce skipping of an exon of the dystrophin pre-mRNA.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15)
Specification