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Multiple exon skipping compositions for DMD

  • US 8,865,883 B2
  • Filed: 03/14/2013
  • Issued: 10/21/2014
  • Est. Priority Date: 10/24/2008
  • Status: Active Grant
First Claim
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1. An isolated antisense oligonucleotide of 20 to 35 nucleotides in length comprising at least 17 contiguous nucleotides of a nucleotide sequence set forth as SEQ ID NO:

  • 2 wherein the oligonucleotide is capable of binding to human dystrophin pre-mRNA to induce exon 44 skipping, and wherein the oligonucleotide comprises a modification to resist degradation of an oligonucleotide;

    RNA heteroduplex by RNase H, and thymine bases (T) are optionally uracil bases (U).

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