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Are Genetic Therapies for Epilepsy Ready for the Clinic?

Street, James S; Qiu, Yichen; Lignani, Gabriele; (2023) Are Genetic Therapies for Epilepsy Ready for the Clinic? Epilepsy Currents 10.1177/15357597231176234. Green open access

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Abstract

In recent years, there has been a significant increase in preclinical studies to test genetic therapies for epilepsy. Some of these therapies have advanced to clinical trials and are being tested in patients with monogenetic or focal refractory epilepsy. This article provides an overview of the current state of preclinical studies that show potential for clinical translation. Specifically, we focus on genetic therapies that have demonstrated a clear effect on seizures in animal models and have the potential to be translated to clinical settings. Both therapies targeting the cause of the disease and those that treat symptoms are discussed. We believe that the next few years will be crucial in determining the potential of genetic therapies for treating patients with epilepsy.

Type: Article
Title: Are Genetic Therapies for Epilepsy Ready for the Clinic?
Open access status: An open access version is available from UCL Discovery
DOI: 10.1177/15357597231176234
Publisher version: https://doi.org/10.1177/15357597231176234
Language: English
Additional information: © The Author(s) 2023. Creative Commons License (CC BY 4.0) This article is distributed under the terms of the Creative Commons Attribution 4.0 License (https://creativecommons.org/licenses/by/4.0/) which permits any use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage).
Keywords: genetic therapies, epilepsy, genetic epilepsies, refractory epilepsy
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/10170757
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