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AES 2022 | Manipulation of epileptic neuronal activity using activity-dependent gene therapy

Around 30% of patients with epilepsy are refractory to pharmacological treatment. Several gene therapy approaches have been suggested, but a limiting factor is that they do not discriminate between neurons involved in circuit pathologies and “healthy” surrounding neurons. Gabriele Lignani, PhD, UCL Queen Square Institute of Neurology, London, UK, discusses a gene therapy strategy developed to target and treat only the neurons involved in the generation of seizures. This tool uses an immediate early gene promoter to drive the expression of a potassium channel, specifically in hyperactive neurons, and only for as long as they exhibit abnormal activity. Pathological hyperactivity triggers the expression of the transgene in transduced neurons, which then reduces their likelihood to fire and release neurotransmitters. Once seizures resolve, the gene therapy tool automatically turns off until excessive activity occurs again. The strategy was tested in animal models of epilepsy and human organoids derived from patients’ stem cells. Neuronal excitability was reduced by seizure-related activity, leading to a persistent antiepileptic effect without interfering with normal behaviors. The next steps in this project will include toxicology studies and the collection of more data to support the initiation of clinical trials. This interview was recorded during an online conference call with The Video Journal of Neurology (VJNeurology).

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GL has equity in a company that aims to bring epilepsy gene therapy to the clinic