On-Demand Gene Therapy Approach Shows Promise for Epilepsy

A gene therapy approach for brain circuit disorders that tamps down the excitability of overactive neurons is reported in Science this week. Many neurodevelopmental and neuropsychiatric circuit disorders are characterized by intermittent episodes of pathological activity. Experimental genetic therapies hold potential for such conditions but are limited by their inability to distinguish between neurons involved in the pathologies and other regions of the brain. In this week's study, a group led by University College London researchers describe a novel strategy based on a transgene that decreases neuronal excitability in hyperactive neurons but becomes inactive once the cells return to normal. The researchers demonstrate the technique in a mouse model of epilepsy, showing that it suppress seizures without affecting normal behaviors. Given that epilepsy is just one of a number of diseases characterized by brain circuit hyperactivity, this approach has wide clinical potential, the study's authors write.