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METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY PRE-mRNA

  • US 20170107512A1
  • Filed: 12/27/2016
  • Published: 04/20/2017
  • Est. Priority Date: 10/26/2007
  • Status: Abandoned Application
First Claim
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1. An antisense oligonucleotide of 25-29 nucleotides in length, comprising the base sequence of 5′

  • -UUUGCCGCUGCCCAAUGCCAUCCUG-3′

    (SEQ ID;

    NO;

         3) or a DNA oligonucleotide thereof, said oligonucleotide comprising a modification and is complementary along its entire length to a sequence within exon 45 of a human dystrophin pre-mRNA, said oligonucleotide being capable of inducing skipping of exon 45 by at least 50%.

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