Modulation of gys1 expression
AssigneesIonis Pharmaceuticals, Inc., The Hospital for Sick Chlidren
InventorsTamar R. Grossman, Susan M. Freier, Berge Minassian, Saija Ahonen
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Abstract
Provided herein are methods, compounds, and compositions for reducing expression of GYS1 in an individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a glycogen storage disease or disorder in an individual in need.
Claims64
A method comprising administering a GYS1-specific inhibitor to an individual.
A method of treating, preventing, delaying the onset, slowing the progression, or ameliorating glycogen storage disease in an individual having, or at risk of having, glycogen storage disease or disorder comprising administering a compound comprising a GYS1-specific inhibitor to the individual, thereby treating, preventing, delaying the onset, slowing the progression, or ameliorating a glycogen storage disease or disorder in the individual.
The method of any preceding claim, wherein the GYS1-specific inhibitor inhibits expression of GYS1 in the individual.
The method of any preceding claim wherein the individual is human.
A method of inhibiting expression or activity of GYS1 in a cell, comprising contacting the cell with a compound comprising a GYS1-specific inhibitor, thereby inhibiting expression or activity of GYS1 in the cell.
The method of any preceding claim, wherein the GYS1-specific inhibitor selected from any of a nucleic acid, a polypeptide, an antibody, and a small molecule.
The method of any preceding claim, wherein the compound comprising a modified oligonucleotide targeting GYS1.
The method of any one of the preceding claims, wherein the compound is administered parenterally.
The method of any of the preceding claims, comprising co-administering the compound and at least one additional therapy.
Use of a compound comprising a GYS1-specific inhibitor for the manufacture or preparation of a medicament for administering to an individual.
Use of a compound comprising a GYS1-specific inhibitor for the manufacture or preparation of a medicament for treating a glycogen storage disease or disorder in an individual.
Use of a compound comprising a GYS1-specific inhibitor for the treatment of a glycogen storage disease or disorder in an individual.
Dependencies are inferred from the claim text.