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Recent progress and challenges in gene therapy for pharmacoresistant focal epilepsy

Kullmann, DM; (2025) Recent progress and challenges in gene therapy for pharmacoresistant focal epilepsy. Revue Neurologique 10.1016/j.neurol.2025.02.008. (In press).

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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.

Type: Article
Title: Recent progress and challenges in gene therapy for pharmacoresistant focal epilepsy
Location: France
DOI: 10.1016/j.neurol.2025.02.008
Publisher version: https://doi.org/10.1016/j.neurol.2025.02.008
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Epilepsy; Gene therapy; Advanced therapies; Preclinical; Pharmacoresistant
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Clinical and Experimental Epilepsy
URI: https://discovery.ucl.ac.uk/id/eprint/10207289
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