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Antisense oligonucleotides for inducing exon skipping and methods of use thereof

  • US 8,455,634 B2
  • Filed: 06/24/2011
  • Issued: 06/04/2013
  • Est. Priority Date: 06/28/2004
  • Status: Active Grant
First Claim
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1. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 17 consecutive nucleotides complementary to an exon 52 target region of the Dystrophin gene designated as annealing site H52A(+17+37), wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 52 skipping, and wherein uracil bases in the antisense oligonucleotide are optionally thymine bases.

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