Modulators of DNM2 expression
AssigneeIonis Pharmaceuticals, Inc.
InventorSusan M. Freier
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Abstract
The present embodiments provide methods, compounds, and compositions useful for inhibiting DNM2 expression, which may be useful for treating, preventing, or ameliorating a disease associated with DNM2.
Claims49
A compound comprising a modified oligonucleotide consisting of 16 to 20 linked nucleosides and having a nucleobase sequence comprising at least 14 contiguous nucleobases of SEQ ID NO:
- 2879 or SEQ ID NO:
- 2123, wherein the modified oligonucleotide comprises at least one of a modified internucleoside linkage and a modified sugar moiety.
A modified oligonucleotide according to the following formula:
- Gks Tks Tks Tds Ads Tds Tds Ads Tds Ads Gds Gds Gds mCks Tks Tk;
- wherein,
- A=an adenine nucleobase,
- mC=a 5-methylcytosine nucleobase,
- G=a guanine nucleobase,
- T=a thymine nucleobase,
- k=a cEt sugar moiety,
- d=a 2′-deoxyribosyl sugar moiety, and
- s=a phosphorothioate internucleoside linkage.
A modified oligonucleotide, wherein the anion form of the modified oligonucleotide has the following chemical structure:
An in vitro method of inhibiting expression of DNM2 in a cell comprising contacting the cell with compound comprising a single-stranded modified oligonucleotide that is 100% complementary to exon 10, intron 1, intron 11, intron 12, intron 13, intron 14, or the 3′-UTR of a DNM2 nucleic acid transcript, thereby inhibiting expression of DNM2 in the cell.
A modified oligonucleotide according to the following chemical structure:
- or a salt thereof.
A modified oligonucleotide according to the following chemical structure:
Dependencies are inferred from the claim text.