Base Editing Rescue of Seizures and SUDEP in SCN8A Developmental Epileptic Encephalopathy
Caeley M Reever, Alexis R. Boscia, Tyler C J Deutsch, Mansi P. Patel, Raquel M. Miralles, Shrinidhi Kittur, Erik J. Fleischel, Atum M. Buo, Matthew S. Yorek, Miriam H. Meisler, Charles R. Farber, Manoj K. Patel
This study uses base editing to correct the SCN8A R1872W mutation in mice, reducing seizures and improving survival and comorbidities.
bioRxiv · Apr 9, 2025 · PreprintBioengineering