Small-molecule activation of TFEB alleviates Niemann–Pick disease type C via promoting lysosomal exocytosis and biogenesis

Niemann–Pick disease type C (NPC) is a devastating lysosomal storage disease characterized by abnormal cholesterol accumulation in lysosomes. Currently, there is no treatment for NPC. Transcription factor EB (TFEB), a member of the microphthalmia transcription factors (MiTF), has emerged as a master...

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Hlavní autoři:	Kaili Du, Hongyu Chen, Zhaonan Pan, Mengli Zhao, Shixue Cheng, Yu Luo, Wenhe Zhang, Dan Li
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	eLife Sciences Publications Ltd 2025-04-01
Edice:	eLife
Témata:	TFEB agonists cholesterol accumulation NPC1 lysosome
On-line přístup:	https://elifesciences.org/articles/103137
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