Cell BiologybioRxiv
Bromodomain proteins as new drug target for an inborn lysosomal defect
M. Parente, A. Barthelemy, C. Tonini…·V. Pallottini
This study investigates bromodomain proteins as a therapeutic target for Niemann-Pick type C disease, showing that JQ1 improves NPC1 levels and reduces cholesterol accumulation in patient-derived fibroblasts.
DOI 10.3390/ijms26125769