NeuroscienceMolecular Therapy AdvancesPeer-reviewed
Neuron-Specific WWOX Gene Therapy Produces Dose-Dependent, Durable Rescue in a Model of WWOX-Related Epileptic Encephalopathy
Mustafa Obeid, Rania Akkawi, Srinivasarao Repudi…·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.
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