The promise of gene therapy for the treatment of epilepsy

Abstract

Gene therapy involves the transfer and expression of a therapeutic gene into a target tissue, and essentially represents an intervention that alters the instruction set of a cell having a dominant effect on the cell phenotype. Gene therapy techniques may provide a realistic therapeutic option for CNS disorders that are difficult to treat with conventional drugs, such as for drug-resistant focal epilepsies. Preclinical studies using a selection of ‘therapeutic’ genes delivered into the rodent brain showed significant reduction of seizures and inhibition of epileptogenesis, thus establishing the proof-of-principle evidence of the potential applicability of this technology to human epilepsy. The promising potential of gene therapy for the treatment of CNS pathologies still requires further investigation to improve the efficacy and the safety of this approach using clinically relevant experimental models. The recent advances in refining gene therapy tools and in methods of gene delivery make this innovative approach a definite possibility for therapeutic intervention.

Keywords::

• animal models of seizures
• anticonvulsant
• cell transplantation
• epileptogenesis
• neurodegeneration
• viral vectors

Financial & competing interests disclosure

Support was provided by Telethon Onlus Foundation and Fondazione Monzino and by Neurologix, Inc. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.