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RNA Modulating Oligonucleotides with Improved Characteristics for the Treatment of Duchenne and Becker Muscular Dystrophy

  • US 20150045413A1
  • Filed: 07/28/2014
  • Published: 02/12/2015
  • Est. Priority Date: 01/27/2012
  • Status: Abandoned Application
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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