NeurosciencebioRxivPreprint
AAV-mediated gene therapy for SLC13A5 citrate transporter disorder rescues epileptic and metabolic phenotypes
Lauren E. Bailey, Morgan K. Schackmuth, Raegan M. Adams…·Rachel M. Bailey
This study shows that AAV-mediated gene therapy in SLC13A5-deficient mice reduces extracellular citrate, normalizes electrophysiologic and sleep issues, and improves seizure resistance.
DOI 10.1101/2025.07.03.663044