Gene therapy-mediated overexpression of wild-type MFN2 improves Charcot-Marie-Tooth disease type 2A
Marine Tessier, Zeinab Hamzé, Nathalie Bonello‐Palot, Nathalie Roëckel, Shahram Attarian, Marc Bartoli, Valérie Delague, Bernard Laurent Schneider, Nathalie Bernard‐Marissal
This study shows that overexpressing wild-type MFN2 via gene therapy improves CMT2A in mice, restoring ER-mitochondria contacts and motor function even after symptom onset.
bioRxiv · Oct 15, 2025 · PreprintNeuroscience