US 11,713,462 B2
Modulation of GYS1 expression
Tamar R. Grossman, La Jolla, CA (US); Susan M. Freier, San Diego, CA (US); Berge Minassian, Toronto (CA); and Saija Ahonen, Toronto (CA)
Assigned to Ionis Pharmaceuticals, Inc., Carlsbad, CA (US); and The Hospital for Sick Children, Toronto (CA)
Filed by Ionis Pharmaceuticals, Inc., Carlsbad, CA (US); and The Hospital for Sick Children, Toronto (CA)
Filed on Dec. 23, 2021, as Appl. No. 17/561,095.
Application 17/561,095 is a division of application No. 16/306,831, granted, now 11,236,339, previously published as PCT/US2017/038109, filed on Jun. 19, 2017.
Claims priority of provisional application 62/430,106, filed on Dec. 5, 2016.
Claims priority of provisional application 62/351,396, filed on Jun. 17, 2016.
Prior Publication US 2022/0112502 A1, Apr. 14, 2022
Int. Cl. C07H 21/02 (2006.01); C07H 21/04 (2006.01); C12N 15/113 (2010.01); A61K 31/7088 (2006.01); A61K 45/06 (2006.01); A61P 3/08 (2006.01); A61P 25/28 (2006.01)
CPC C12N 15/1137 (2013.01) [A61K 31/7088 (2013.01); A61K 45/06 (2013.01); A61P 3/08 (2018.01); A61P 25/28 (2018.01); C12N 15/113 (2013.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/3341 (2013.01); C12N 2310/341 (2013.01); C12N 2320/30 (2013.01)] 17 Claims
 
1. A method of treating Lafora disease in an individual having Lafora 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 at least 95% complementary to an equal length portion of the nucleobase sequence of SEQ ID NO: 6, wherein at least one internucleoside linkage of the modified oligonucleotide is a phosphorothioate internucleoside linkage, and wherein Lafora bodies are reduced in a cell in the individual, thereby treating the Lafora disease in the individual.