GYS1 Antisense Therapy Prevents Disease-Driving Aggregates and Epileptiform Discharges in a Lafora Disease Mouse Model

GYS1 Antisense Therapy Inhibits Pathogenic Aggregates and Epileptiform Discharges in a Mouse Model of Lafora Disease Background and Objectives Lafora disease (LD) is a devastating autosomal recessive genetic disorder characterized by epilepsy and rapidly progressive dementia in adolescence. The disease primarily involves mutations in the EPM2A or E...