Current prospects and challenges for epilepsy gene therapy

This review addresses the state of gene therapy research for the treatment of epilepsy. Preclinical studies have demonstrated the anti-seizure efficacy of viral vector-based gene transfer through the use of a variety of strategies — from modulating classic neurotransmitter systems to targeting or overexpressing of neuropeptide receptors in seizure-specific brain regions. While these studies provide substantive proof of principle for viral vector gene therapy, future studies must address the challenges of vector immunity, cellular specificity and effective global delivery. As these issues are resolved, viral vector gene therapy should significantly impact the treatment of intractable epilepsy.