Preclinical validation of AAV9-TECPR2 gene therapy in a novel knock-in model of TECPR2-related disorder
Bruna Lenfers Turnes, Peter Casey-Caplan, Leo Mejia, Tiffany Berry, Julie Zhao, Francesco Villa, Emma Cropper, Maryam Arab, Biyao Zhang, Darred Surin, Darius Ebrahimi‐Fakhari, Silmara de Lima, Alan S. Kopin, Nick J Andrews, Nathaniel Hodgson, Michela Fagiolini
This study develops a TECPR2 knock-in mouse model and shows that AAV9-mediated gene therapy improves mechanosensory function, gait, and startle responses in neonatal mice with TECPR2 deficiency.
bioRxiv · Mar 25, 2026 · PreprintNeuroscience