%0 Journal Article
%T TFEB在溶酶体贮积症中的调控作用及研究进展<br> The Regulatory Role and Re-search Progress of TFEB in Lysosomal Storage Disease
%A 程士雪
%A 赵梦丽
%J Bioprocess
%P 1-6
%@ 2164-5582
%D 2023
%I Hans Publishing
%R 10.12677/BP.2023.131001
%X 溶酶体贮积症是一种罕见的遗传缺陷疾病，由于编码溶酶体水解酶、膜转运蛋白或运输蛋白的基因突变，最终导致细胞功能障碍。转录因子EB (TFEB)是自噬和溶酶体生物发生的主要调节因子，可促进溶酶体合成及溶酶体功能的恢复。本文总结了TFEB的调节机制及TFEB的小分子激动剂在溶酶体贮积症的治疗作用及研究进展。<br />
Lysosomal storage disorder is a rare genetic defect disorder that ultimately leads to cell dysfunction due to mutations in genes encoding lysosomal hydrolases, membrane transporters, or transport proteins. The transcription factor EB (TFEB) is a major regu-lator of autophagy and lysosomal biogenesis, which promotes lysosomal synthesis and the recovery of lysosomal function. This article summarizes the regulatory mechanism of TFEB and the thera-peutic effect and research progress of small molecule agonists of TFEB in lysosomal storage disease.
%K 溶酶体贮积症，转录因子EB，溶酶体<br> Lysosomal Storage Disorders
%K TFEB
%K Lysosome
%U http://www.hanspub.org/journal/PaperInformation.aspx?PaperID=62109