Genetic medicines for epilepsy: unlocking new avenues for seizure control

Epilepsy affects millions of people globally and is marked by unpredictable seizures due to excessive brain activity. These seizures not only vary widely in brain origin and severity but can also be associated with a range of factors - from head injuries to infections to genetic causes. Although antiseizure medications provide effective seizure control for many patients, approximately 30% experience drug-resistant epilepsy, with syndromic forms such as Lennox-Gastaut and Dravet syndrome posing significant therapeutic challenges. In addition, current pharmacological treatments are often associated with significant side effects and typically do not address the underlying pathophysiology. Gene therapies and genetic medicines are groundbreaking treatment modalities that enable direct targeting of disease mechanisms and associated genes. After FDA approval of the very first gene therapy in 2017 and the discovery of CRISPR-based gene editing, the field has rapidly expanded offering new hope for epilepsy treatment. This review highlights the latest advancements and therapeutic approaches for genetic medicines and explores their potential to transform the therapeutic landscape of epilepsy.