Review article

Gene therapy in refractory epilepsy

Paula Polenus ; School of Medicine, University of Zagreb, Zagreb, Croatia

Abstract

Epilepsy is one of the most common neurological disorders affecting a significant portion of the world population. Approximately 30% of epilepsy cases are not effectively treated with anti-seizure medications (ASMs), leading to pharmacoresistant epilepsies, which affect up to 0.3% of the global population. Current therapies available to patients with refractory epileptic seizures include electrical stimulation, or surgery, with approximately two-thirds of patients achieving a seizure-free state. Gene therapy emerges as a novel and innovative approach, presenting a paradigm shift in the treatment landscape. Various treatment strategies tested in in vivo models are discussed, including overexpression of potassium channels, kainate receptor downregulation using RNA interference and others. As the field progresses, ongoing research and upcoming clinical trials are expected to contribute to our understanding of gene therapy's role in revolutionising the management of pharmacoresistant epilepsy.

Keywords

Adeno-associated virus (AAV); gene therapy; Lentivirus; neurological disorders; refractory epilepsy; temporal lobe epilepsy (TLE); viral vectors

Hrčak ID:

353436

URI

https://hrcak.srce.hr/353436

Publication date:

20.12.2024.

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