A gene therapy approach for focal epilepsy based on GABA\(_A\) receptor overexpression
Martina Bonfanti, Alessandro Gaeta, Lilian Juliana Lissner, Stefano Cattaneo, Gabriele Ruffolo, Eleonora Palma, Michele Simonato, Barbara Bettegazzi · FENS Forum 2024
Epilepsy is a chronic neurological disorder characterized by recurrent seizures. Despite the availability of many antiepileptic drugs (AEDs), 30% of the patients are resistant to standard therapies. GABAA receptor (GABAAR) expression and function are altered in the epileptic tissue, resulting in impaired inhibition and reduced sensitivity to GABAergic AEDs. Therefore, we are developing a gene therapy strategy for focal drug-resistant epilepsy based on the overexpression of specific GABAAR subunits in hippocampal excitatory neurons, aiming at increasing network inhibition and enhancing the effe
ePoster · Neuroscience