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

神经退行性溶酶体贮积症的分子病理生理学机制仍不清楚。在本研究中，我们发现在这种疾病的培养神经元模型中，自噬降解和线粒体功能受损以及组蛋白去乙酰化酶的失调。我们还研究了自噬和组蛋白去乙酰化酶抑制剂在该模型细胞中的治疗效果。本研究结果为进一步开发新型分子靶向治疗溶酶体贮积病神经变性提供了重要的参考。

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.