Neuron-Specific WWOX Gene Therapy Produces Dose-Dependent, Durable Rescue in a Model of WWOX-Related Epileptic Encephalopathy
Mustafa Obeid, Rania Akkawi, Srinivasarao Repudi, Prince Kumar Singh, Baraa Abudiab, Takwa Jebara, Allyson Berent, Thomas Brennan, Yael Weiss, Tawfeeq Shekh‐Ahmad, Rami I. Aqeilan
This study identifies optimal parameters for neuron-targeted WWOX gene therapy in a mouse model of WWOX-related epileptic encephalopathy, showing durable rescue with a defined therapeutic window and dose.
Molecular Therapy Advances · Jun 19, 2026Peer-reviewedNeuroscience