Bromodomain proteins as new drug target for an inborn lysosomal defect
M. Parente, A. Barthelemy, C. Tonini, S. Caputo, A. Sacchi, S. Leone, M. Segatto, F. W. Pfrieger, 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.
bioRxiv · Dec 19, 2023Cell Biology