Selective antisense compounds and uses thereof
AssigneeIonis Pharmaceuticals, Inc.
InventorsMichael Oestergaard, Punit P. Seth, Eric E. Swayze
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Abstract
The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount, activity, or expression of the target nucleic acid in a cell. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.
Claims24
A compound comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides, wherein the nucleobase sequence of the modified oligonucleotide comprises at least 8 contiguous nucleobases of the nucleobase sequence recited in SEQ ID NO:
- 2, wherein the modified oligonucleotide has a sugar motif selected from ekek-d9-keke, kekk-d8-keke, ekkk-d8-kke, ekk-d8-kkke, ekekk-d8-keke, ekek-d8-kkeke, eekk-d8-kkeee, and eeekk-d8-kkee, wherein k is a cEt sugar moiety, e is a 2′-OCH2CH2OCH3 ribosyl sugar moiety, and d is a 2′-deoxyribosyl sugar moiety.
Dependencies are inferred from the claim text.