溶酶体贮积症(lysosomal storage diseases,LSDs)及神经元变性疾病均可累及神经系统,出现相应症状体征,它们共同发病机制为溶酶体功能障碍导致代谢产物异常蓄积,使神经元发生变性、凋亡等。TFEB基因作为溶酶体的主控基因,在机体病理生理过程中起广泛的作用,可促进溶酶体合成及溶酶体功能的恢复,为以溶酶体功能障碍为发病机制的神经系统疾病提供一种新的治疗方法。 Lysosomal storage diseases (LSDs) and neurodegenerative diseases can affect the nervous system, the corresponding symptoms and signs, their common pathogenesis of lysosomal dysfunction lead to abnormal accumulation of metabolites, degeneration of neurons , Apoptosis and so on. TFEB gene as a lysosomal control gene plays a broad role in the pathophysiology of the body can promote lysosomal synthesis and lysosomal function recovery for the pathogenesis of lysosomal dysfunction as a neurological disease Provide a new treatment method.