Gene therapy for epilepsies

Abstract

Epilepsy is an excellent indication for gene therapy due to a significant unmet need, which is the high percentage of patients with symptoms that remain inadequately relieved by the current available treatments. This chapter provides an up-to-date review on the preclinical studies assessing the potential of gene therapy for epilepsy. We also provide here a set of procedures that can be used as a starting point to evaluate potential therapeutic candidates for epilepsy gene therapy. The techniques described here include adeno-associated viral vector production, genomic titering of the vector, stereotaxic neurosurgery for intracerebral vector administration, and the kainic acid seizure model.