Recent progress and challenges in gene therapy for pharmacoresistant focal epilepsy - 30/03/25
Abstract |
Pharmacoresistant focal epilepsy represents a major unmet need. Recent years have seen several gene therapy strategies validated mainly in rodent models of temporal lobe epilepsy, and some of these have been de-risked for clinical trials. This review considers some of the challenges in progressing from experimental models to the clinic. Among these are identifying promising promoter-transgene combinations, establishing safe and efficacious doses, achieving optimal delivery, and extrapolating across different aetiologies.
Le texte complet de cet article est disponible en PDF.Keywords : Epilepsy, Gene therapy, Advanced therapies, Preclinical, Pharmacoresistant
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