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Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
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机译:具有改良特性的Rna调节寡核苷酸,用于治疗杜兴氏和贝克氏肌营养不良症
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摘要
Discloses 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 for use in the treatment of Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
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