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RNA Modulating Oligonucleotides with Improved Characteristics for the Treatment of Neuromuscular Disorders

  • US 20150148404A1
  • Filed: 10/23/2014
  • Published: 05/28/2015
  • Est. Priority Date: 04/23/2012
  • Status: Active Grant
First Claim
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1. An oligonucleotide comprising a 2′

  • -O-methyl RNA nucleotide residue, having a backbone wherein at least one phosphate moiety is replaced by a phosphorothioate moiety, and comprising one or more 5-methylpyrimidine and/or one or more 2,6-diaminopurine bases, wherein said oligonucleotide is able to hybridize to a repetitive element having as its repetitive nucleotide unit a repetitive nucleotide unit selected from the list consisting of (CAG)n, (GCG)n, (CGG)n, (GAA)n, (GCC)n, (CCG)n, (AUUCU)n, (GGGGCC)n and (CCUG)n.

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