Compounds and methods for the modulation of proteins
AssigneeIonis Pharmaceuticals, Inc.
InventorsStanley T. Crooke, Xue-hai Liang, Wen Shen
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Abstract
In certain embodiments, the present disclosure provides compounds and methods of increasing the amount or activity of a target protein in a cell. In certain embodiments, the compounds comprise a translation suppression element inhibitor. In certain embodiments, the translation suppression element inhibitor is a uORF inhibitor. In certain embodiments, the uORF inhibitor is an antisense compound.
Claims15
A method of increasing the amount of a target protein in a cell, comprising contacting the cell with an antisense compound comprising a modified oligonucleotide, wherein the target protein is encoded by a target transcript comprising at least one translation suppression element in the 5′ untranslated region of the target transcript that is a uORF, wherein the modified oligonucleotide is complementary to a target site comprising the uORF;
- and thereby increasing the amount of the target protein in the cell.
Dependencies are inferred from the claim text.