Small-molecule Activation of TFEB Alleviates Niemann-Pick Disease Type C via Promoting Lysosomal Exocytosis and Biogenesis
Kaili Du, Hongyu Chen, Zhaonan Pan, Mengli Zhao, Shixue Cheng, Yu Luo, Wenhe Zhang, Dan Li
This study shows that activating TFEB with sulforaphane reduces cholesterol accumulation in NPC1 models by enhancing lysosomal exocytosis and biogenesis.
eLife · Apr 4, 2025Peer-reviewedMolecular Biology