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Modulation of huntingtin expression

  • US 9,273,315 B2
  • Filed: 10/30/2014
  • Issued: 03/01/2016
  • Est. Priority Date: 09/11/2009
  • Status: Active Grant
First Claim
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1. A single-stranded modified oligonucleotide consisting of 20 linked nucleosides and having:

  • a gap segment consisting of ten linked deoxynucleosides;

    a 5′

    wing segment consisting of five linked nucleosides; and

    a 3′

    wing segment consisting of five linked nucleosides;

    wherein the gap segment is positioned between the 5′

    wing segment and the 3′

    wing segment;

    wherein each nucleoside of each wing segment comprises a 2′

    O-methoxyethyl sugar;

    wherein the internucleoside linkages within the gap segment, the linkages connecting the gap segment to the 5′ and

    3′

    wing segments, and the linkages for the 5′

    -most and 3′

    -most nucleosides of each wing segment are all phosphorothioate linkages; and

    the internucleoside linkages connecting the rest of the nucleosides of both the 5′ and

    3′

    wing segments are phosphodiester linkages; and

    wherein the nucleobase sequence of the oligonucleotide consists of the sequence recited in SEQ ID NO;

    22,or a pharmaceutically acceptable salt thereof.

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