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Splice-region antisense composition and method

  • US 8,436,163 B2
  • Filed: 08/26/2011
  • Issued: 05/07/2013
  • Est. Priority Date: 05/04/2000
  • Status: Expired due to Term
First Claim
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1. An antisense compound composed of a 2′

  • -O-methyl modified backbone and a base sequence of 12 to 25 nucleotide bases which is complementary to a target region within one exon of multiple exons of a preprocessed mRNA encoding a human protein, wherein the 5′

    -end of the target region is 6 or 7 bases downstream of a normal splice acceptor site in said preprocessed mRNA.

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