Antisense nucleic acids

  • US 10,662,217 B2
  • Filed: 12/12/2019
  • Issued: 05/26/2020
  • Est. Priority Date: 09/01/2010
  • Status: Active Grant
First Claim
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1. A method of treating a DMD patient comprising intravenously administering to said patient an oligomer comprising:

  • a) a phosphorodiamidate morpholino oligomer (PMO) that is 100% complementary to the 36th to the 60th nucleotides from the 5′

    end of the 53rd exon in a human dystrophin pre-mRNA, wherein the 53rd exon in said human dystrophin pre-mRNA consists of a nucleotide sequence corresponding to SEQ ID NO;

    1, wherein said PMO hybridizes to said human dystrophin pre-mRNA with Watson-Crick base pairing, wherein the phosphorodiamidate morpholino monomers of said PMO have the formula;

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