NPC1-mTORC1 signaling Couples Cholesterol Sensing to Organelle Homeostasis and is a Targetable Pathway in Niemann-Pick type C
O. B. Davis, H. R. Shin, C.-Y. Lim, E. Y. Wu, M. Kukurugya, C. F. Maher, R. M. Perera, P. Ordonez, R. Zoncu
This study identifies mTORC1 hyperactivation as a downstream driver of lysosomal dysfunction in NPC, showing that its inhibition can restore proteolysis and mitigate mitochondrial dysfunction.
bioRxiv · Aug 2, 2020Cell Biology