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Antisense nucleic acids

DC CAFC
  • US 10,407,461 B2
  • Filed: 03/26/2019
  • Issued: 09/10/2019
  • Est. Priority Date: 09/01/2010
  • Status: Active Grant
First Claim
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1. A phosphorodiamidate morpholino oligomer (PMO) antisense oligomer that causes skipping of the 53rd exon in a human dystrophin pre-mRNA, consisting of a 25-mer oligomer that is 100% complementary to the target sequence 5′

  • -GAACACCUUCAGAACCGGAGGCAAC-3′

    (SEQ ID NO;

         124) of said human dystrophin pre-mRNA, wherein said PMO antisense oligomer hybridizes to said target sequence with Watson-Crick base pairing under physiological conditions, wherein each phosphorodiamidate morpholino monomer of said PMO antisense oligomer has the formula;

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