MAPT Splicing Modulators as a Therapeutic Strategy for Tauopathies
M. Catarina Silva, Hannah Lindmeier, Paolo Pigini, Jennifer Laughlin, Yong Yu, Christie Morrill, Michael A. Arnold, Scott J. Barraza, Khalil Saadipour, Monal Dieterich, Angela Minnella, Kausiki Datta, Nanjing Zhang, Jana Narasimhan, Christopher R. Trotta, , Elisabetta Morini
Discovery of SMCs that correct MAPT splicing, reduce 4R tau, and rescue pathology in patient-derived neuronal and in vivo models of 4R tauopathies.
bioRxiv · May 6, 2025 · PreprintNeuroscience