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Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene

  • US 10,036,014 B2
  • Filed: 11/18/2014
  • Issued: 07/31/2018
  • Est. Priority Date: 06/26/2009
  • Status: Active Grant
First Claim
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1. A synthetic, modified oligonucleotide of 10 to 30 nucleotides in length comprising at least one modification wherein the at least one modification is selected from:

  • at least one modified sugar moiety;

    at least one modified internucleotide linkage;

    at least one modified nucleotide, and combinations thereof;

    wherein said oligonucleotide is an antisense compound which is at least 90% complementary to and specifically hybridizes to a natural antisense polynucleotide of DSCR1 gene selected from the group consisting of SEQ ID NO;

    3, 4 5 or 6 and upregulates the function and/or expression of said DSCR1 gene in vivo orin vitro as compared to a normal control.

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