Gene Therapy in Epilepsy

Abstract

The generation of viral vectors, such as adeno-associated virus (AAV) and lentivirus, which are capable of stable transduction of neurons, offers an attractive strategy for introducing novel genes into the brain, resulting in a long-lasting production of specific proteins. An alternative approach to achieving transgene expression in brain is to graft cells that are genetically engineered to produce neuroactive substances. Neuroactive peptides, adenosine, and γ-aminobutyric acid, are agents that can be delivered by gene and cell therapy with potential utility in epilepsy therapy.