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 gamma-aminobutyric acid, are agents that can be delivered by gene and cell therapy with potential utility in epilepsy therapy.