Compounds and methods for improved cellular uptake of antisense compounds
AssigneeIonis Pharmaceuticals, Inc.
InventorsErich Koller, C. Frank Bennett
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Abstract
The present invention provides method of increasing the efficacy and potency of antisense compounds. In certain embodiments, the invention provides methods for improved cellular uptake.
Claims21
-168. (canceled)
A method of sensitizing a cell for antisense modulation comprising, reducing the amount or activity of at least one nucleic acid transcript in the cell, wherein the at least one nucleic acid transcript in the cell is a Lip5, Rab27A, Rab27B, SYTL4, SLAC2B, or AP2M1 transcript;
- and thereby sensitizing the cell for antisense modulation;
- and contacting the cell with at least one antisense compound complementary to a target nucleic acid other than Lip5, Rab27, SYTL4, SLAC2B, or AP2M1.
A method of sensitizing a cell for antisense modulation comprising, reducing the amount or activity of at least one ESCRT transcript in the cell, wherein the at least one ESCRT transcript in the cell is a Vps28, Tsg101, Vps37, Mvb12a, Mvb12b, Hrs, Alix, Vps2, Vps4, Vps20, Vps22, Vps24, Vps25, Vps32, Vps36, Vps60, lst1, or Did2 transcript, and thereby sensitizing the cell for antisense modulation;
- and contacting the cell with at least one non-ESCRT antisense compound, wherein the non-ESCRT antisense compound is complementary to a target nucleic acid other than a Vps28, Tsg101, Vps37, Mvb12a, Mvb12b, Hrs, Alix, Vps2, Vps4, Vps20, Vps22, Vps24, Vps25, Vps32, Vps36, Vps60, or Did2 transcript.
Dependencies are inferred from the claim text.