Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals
AssigneeIonis Pharmaceuticals, Inc.
InventorsXue-hai Liang, Stanley T. Crooke
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Abstract
The present invention provides compounds and methods for modulating target nucleic acids found in organelles or sub-organelles of cells. The invention includes, but is not limited to compounds and methods that modulate target nucleic acids in a sub-nuclear organelle, such as the nucleolus and/or a cajal body. In certain embodiments, the cell is in an animal.
Claims17
A method of reducing the amount of a target snoRNA in a cell in an animal comprising administering to the animal a pharmaceutical composition comprising a single-stranded antisense compound comprising a modified oligonucleotide complementary to the target snoRNA;
- and thereby reducing the amount of the target snoRNA in the cell in the animal;
- wherein the modified oligonucleotide comprises:
- a 5′-region consisting of 1 to 7 modified nucleosides;
- a 3′-region consisting of 1 to 7 modified nucleosides;
- and
- a central region consisting of 5 to 28 deoxyribonucleosides;
- and
- wherein the animal is a mammal
- and wherein the target snoRNA is a U16 snoRNA, a U23 snoRNA, or a U50 snoRNA.
Dependencies are inferred from the claim text.