Compounds and methods for modulation of DUX4
AssigneesIonis Pharmaceuticals, Inc., Fred Hutchinson Cancer Research Center
InventorsFrank Rigo, Stephen J. Tapscott
Life of this patent
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Abstract
The present disclosure pertains generally to double-stranded small interfering RNAs that modulate gene expression for use in research, diagnostics, and/or therapeutics. In certain embodiments, the present disclosure provides double-stranded small interfering RNAs that modulate DUX4 gene expression. In certain embodiments, the present disclosure provides methods of inhibiting DUX4 gene expression by contacting a cell with double-stranded small interfering RNAs.
Claims17
A double-stranded small interfering RNA, comprising a sense strand and an antisense strand, wherein the antisense strand of the double-stranded small interfering RNA has a nucleobase sequence complementary to at least 12 contiguous nucleobases of:
- an equal length portion of nucleobases 5,195-5,220 of SEQ ID NO:
- 1;
- an equal length portion of nucleobases 5,467-5,491 of SEQ ID NO:
- 1;
- or
- an equal length portion of nucleobases 5,815-5,840 of SEQ ID NO:
- 1;
- and
- wherein said sense strand is at least 85% complementary to said antisense strand;
- wherein said sense strand is at least 85% identical to an equal length portion of SEQ ID NO:1;
- and
- wherein said sense strand comprises at least one modified nucleoside.
A double-stranded small interfering RNA, comprising a sense strand and an antisense strand, wherein the antisense strand of the double-stranded small interfering RNA has a nucleobase sequence comprising at least 12 contiguous nucleobases of any of the nucleobase sequences of SEQ ID NOs:
- 13, 21, 23, 27, 33, 39, or 43;
- and wherein said sense strand is at least 85% complementary to said antisense strand, wherein said antisense strand is at least 85% complementary to SEQ ID NO:
- 1, and wherein said sense strand comprises at least one modified nucleoside.
Dependencies are inferred from the claim text.