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Treatment of Frataxin (FXN) related diseases by inhibition of natural antisense transcript to FXN

  • US 9,902,959 B2
  • Filed: 02/02/2017
  • Issued: 02/27/2018
  • Est. Priority Date: 06/09/2011
  • Status: Active Grant
First Claim
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1. A pharmaceutical composition comprising a single-stranded 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 intemucleotide linkage;

    at least one modified nucleotide, and combinations thereof;

    wherein said oligonucleotide is an antisense compound which specifically hybridizes to a natural antisense polynucleotide of a Frataxin (FXN) polynucleotide having SEQ ID NO;

    2 and upregulates the function and/or expression of a Frataxin (FXN) gene in vivo or in vitro as compared to a normal control and a pharmaceutically acceptable excipient.

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