Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy

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.