Use of guaiacol for the prevention and treatment of glycogen storage disease
AssigneesThe Trustees of Columbia University in the City of New York, Hadasit Medical Research Services and Development Ltd., Ramot at Tel-Aviv University Ltd.
InventorsHasan Orhan Akman, Salvatore Dimauro, Or Kakhlon, Miguel Enrique Weil
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Abstract
The current invention is a method of preventing and treating certain diseases by preventing the synthesis and/or breakdown of glycogen by the administration of an agent, guaiacol. Diseases that can be prevented and treated by the administration of guaiacol include but are not limited to glycogen storage disease type IV (GSD-IV), adult polyglucosan body disease (APBD), and Lafora disease (LD). The invention also includes methods and tools for screening for agents that prevent and treat these diseases as well as basic research, specifically in the form of cells and cell lines that produce detectable polyglucosan.
Claims10
A method for ameliorating a disease or a disorder characterized by polyglucosan accumulation or abnormal glycogen accumulation, wherein said disease or disorder is selected from the group consisting of types I-IX glycogen storage disease, adult polyglucosan body disease (APBD), and Lafora disease in a subject in need thereof, the method comprising administering to said subject a therapeutically effective amount of guaiacol.
A method for inhibiting at least one of glycogen synthase (GYS) 1 and GYS2 in a subject afflicted with a disease or a disorder characterized by polyglucosan accumulation or abnormal glycogen accumulation, the method comprises administering to said subject an effective amount of guaiacol, thereby inhibiting at least one of GYS1 and GYS2 in the subject afflicted with a disease or a disorder characterized by polyglucosan accumulation or abnormal glycogen accumulation.
Dependencies are inferred from the claim text.