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A Compact GAD67 Promoter Enables Inhibitory Neuron-Targeted Gene Therapy for Drug-Resistant Epilepsy

Jul 08, 2026

Researchers developed the compact GAD67 promoter (cmGAD67), which enables selective gene expression in inhibitory neurons. AAV-mediated GAD65 delivery suppressed seizures in multiple mouse models of epilepsy, establishing a promising platform for gene therapy of drug-resistant epilepsy and related neurological disorders.