Pharmacological BET Protein Inhibition Reverts Niemann-Pick Type C Disease-Associated Changes in Human iPSC-Derived Cortical Neurons
Federica Benigni, Martina Parente, Chiara Marioli, Rossella Borghi, Nicolò Cicolani, Amélie Barthelemy, Stefania Petrini, Marco Tartaglia, Frank W. Pfrieger, Valentina Pallottini, Claudia Compagnucci
This study uses iPSC-derived neurons from NPC1 patients to show that BET inhibition with JQ1 improves neuronal viability and reduces cholesterol accumulation.
bioRxiv · Sep 3, 2026 · PreprintNeuroscience