NeurosciencebioRxivPreprint
Pharmacological BET Protein Inhibition Reverts Niemann-Pick Type C Disease-Associated Changes in Human iPSC-Derived Cortical Neurons
Federica Benigni, Martina Parente, Chiara Marioli…·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.
DOI 10.64898/2026.08.31.747771