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LGI1 Gene Therapy for Epilepsy

Overexpression of LGI1 using an AAV vector can reduce neuronal and network excitability seen in drug-resistant focal epilepsy

Background

Epilepsy is a widespread serious neurological disorder that impacts 1% of the global population. It is estimated that 30% of these individuals are refractory to pharmacological treatment, and surgical removal of the focal brain area remains the primary option for achieving seizure control, albeit feasible for only 5-10% of patients.

Gene therapy, therefore, presents a promising alternative of pharmaco-resistant focal epilepsy, and researchers have demonstrated the effectiveness of viral overexpression of the potassium channel Kv1.1 to reduce neuronal excitability. This approach offers a potential solution that could avoid the adverse consequences associated with surgical resection.

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