Targeting Gys1 with AAV‐SaCas9 Decreases Pathogenic Polyglucosan Bodies and Neuroinflammation in Adult Polyglucosan Body and Lafora Disease Mouse Models
Emrah Gumusgoz, Dikran R. Guisso, Sahba Kasiri, Jun Wu, Matthew Dear, Brandy Verhalen, Silvia Nitschke, Sharmistha Mitra, Felix Nitschké, Berge A. Minassian
Topics & Concepts
Lafora diseaseNeuroinflammationNeurodegenerationMedicineProgressive myoclonus epilepsyBiologyDiseaseNeuroscienceInternal medicineGeneticsPhosphorylationPhosphataseGlycogen Storage Diseases and MyoclonusGenetics and Neurodevelopmental DisordersAutoimmune Neurological Disorders and Treatments