Development of novel therapeutic strategies for neurodegenerative lysosomal storage diseases by ameliorating autophagic degradation and brain-specific peptides

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Molecular pathophysiology of neurodegenerative lysosomal storage disease ia still largely unclear. In this study, we found impairments of autophagy degradation and mitochondrial function and dysregulation of histone deacetylases in cultured neuronal model of this disease. We also examined the therapeutic effects of inhibitors for autophagy and histone deacetylase in this model cells. The obtained results gave us important insight into the development of novel molecular targeting therapy for neurodegeneration of lysosomal storage diseases in the future.