Cell BiologyInternational Journal of Molecular SciencesPeer-reviewed
Bromodomain proteins as new drug target for an inborn lysosomal defect
Martina Parente, Amélie Barthelemy, Claudia Tonini…·Valentina 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.
3 citations