Treatment of Frataxin (FXN) related diseases by inhibition of natural antisense transcript to FXN
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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Abstract
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Frataxin (FXN), in particular, by targeting natural antisense polynucleotides of Frataxin (FXN). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of FXN.
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14 Claims
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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. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13)
- at least one modified sugar moiety;
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14. 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 intemucleotide linkage;
at least one modified nucleotide, and combinations thereof;
wherein said oligonucleotide is an antisense compound which is 100% complementary to and specifically hybridizes to a complementary target region on nucleotides 1-88 and nucleotides 108-454 of 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.
- at least one modified sugar moiety;
Specification