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Treatment of lipid transport and metabolism gene related diseases by inhibition of natural antisense transcript to a lipid transport and metabolism gene

  • US 10,604,755 B2
  • Filed: 07/31/2015
  • Issued: 03/31/2020
  • Est. Priority Date: 05/06/2009
  • Status: Active Grant
First Claim
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1. A compound comprising a single stranded synthetic, modified antisense RNA oligonucleotide of 19 to 21 nucleotides in length or an optionally modified double stranded siRNA comprising an oligonucleotide of 19 to 30 nucleotides in length wherein said modifications are selected from:

  • at least one modified sugar moiety;

    at least one modified internucleotide linkage;

    at least one modified nucleotide, and combinations thereof;

    wherein said siRNA oligonucleotide and said single stranded antisense RNA oligonucleotide are each 100% complementary with and specifically hybridize to a target region of a natural antisense polynucleotide of a lipid transport and metabolism gene that is an ABCA1 polynucleotide wherein said natural antisense polynucleotide is SEQ ID NO;

    8, and where the antisense RNA oligonucleotide and the siRNA oligonucleotide upregulate the function and/or expression of the ABCA1 polynucleotide in vivo or in vitro as compared to a normal control, and wherein the target region in SEQ ID NO;

    8 consists of nucleotides 1-245 and 799-1169, and further wherein the siRNA oligonucleotide and the single stranded oligonucleotide comprise a nucleobase sequence selected from the group consisting of SEQ ID NOS;

    27, 28, 29, 31, 32, 33, 35, 38, and 39 wherein each thymine base may be replaced by a uracil base.

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