Exogenous Flupirtine as Potential Treatment for CLN3 Disease

摘要: CLN3 disease is a fatal neurodegenerative disorder affecting children. Hallmarks include brain atrophy, accelerated neuronal apoptosis, and ceramide elevation. Treatment regimens are supportive, highlighting the importance of novel, disease-modifying drugs. Flupirtine its new allyl carbamate derivative (compound 6) confer neuroprotective effects in CLN3-deficient cells. This study lays groundwork for investigating beneficial Cln3Δex7/8 mice. WT/Cln3Δex7/8 mice received flupirtine/compound 6/vehicle 14 weeks. Short-term effect flupirtine or compound 6 was tested using battery behavioral testing. For flupirtine, gene expression profiles, astrogliosis, cell counts were determined. improved neurobehavioral parameters open field, pole climbing, Morris water maze tests Several anti-apoptotic markers synthesis/degradation enzymes dysregulated reduced astrogliosis hippocampus motor cortex male female increased The newly synthesized showed promising results field climbing. In conclusion, behavioral, neuropathological biochemical mice, paving way potential therapies disease.