Exploring gene therapy for developmental and epileptic encephalopathies (DEEs): possibilities or promises?

As advances in neurobiology have accelerated over the past decade, our understanding of pediatric neurodevelopmental pathologies has been transformed, revealing how intricate the developing brain can be. Among the most challenging of these conditions lies the developmental and epileptic encephalopathies (DEEs) where severe symptoms collide with limited therapeutic options. Defined as early-onset epilepsies in which developmental delay and cognitive impairment results from the underlying etiology, DEEs often combine frequent drug-resistant seizures with developmental slowing or regression. Population-based data estimate a...