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Reduced size self-delivering RNAi compounds

  • US 9,080,171 B2
  • Filed: 03/24/2011
  • Issued: 07/14/2015
  • Est. Priority Date: 03/24/2010
  • Status: Active Grant
First Claim
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1. An sd-rxRNA2 comprisinga guide strand and a passenger strand, wherein the sd-rxRNA®

  • includes a double stranded region and a single stranded region, wherein the double stranded region is from 8-15 nucleotides long, wherein the single stranded region is at the 3′

    end of the guide strand and is 4-12 nucleotides long, wherein the single stranded region contains 3, 4, 5, 6, 7, 8, 9, 10, 11 or 12 phosphorothioate modifications,wherein at least 40% of the nucleotides of the isolated double stranded nucleic acid molecule are modified, wherein at least two Us and/or Cs include a hydrophobic modification selected from the group consisting of a thiophene, ethynyl, isobutyl benzyl and imidazole modification, and wherein the hydrophobic modifications are located on positions 4 or 5 of the Us and/or Cs.

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