Modulation of GYS1 expression
AssigneesIonis Pharmaceuticals, Inc., The Hospital for Sick Children
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.
Claims21
A method of treating adult polyglucosan body disease in an individual having adult polyglucosan body disease comprising administering to the individual a compound comprising a modified oligonucleotide consisting of 15 to 30 nucleosides, wherein the modified oligonucleotide has a nucleobase sequence complementary to an equal length portion of the nucleobase sequence of any one of SEQ ID NOs:
- 2-6, and wherein at least one internucleoside linkage of the modified oligonucleotide is a phosphorothioate internucleoside linkage, thereby treating the adult polyglucosan body disease in the individual.
A method of inhibiting expression or activity of GYS1 in a cell of an individual with adult polyglucosan body disease comprising contacting the cell with a compound comprising a modified oligonucleotide consisting of 15 to 30 nucleosides, wherein the modified oligonucleotide has a nucleobase sequence complementary to an equal length portion of the nucleobase sequence of any one of SEQ ID NOs:
- 2-6, and wherein at least one internucleoside linkage of the modified oligonucleotide is a phosphorothioate internucleoside linkage, thereby inhibiting expression or activity of GYS1 in the cell.
Dependencies are inferred from the claim text.