用于癫痫的基因疗法准备好进入临床了吗？
Are Genetic Therapies for Epilepsy Ready for the Clinic?
作者信息
Street James S, Qiu Yichen, Lignani Gabriele
机构信息
Department of Clinical and Experimental Epilepsy, UCL Queen Square Institute of Neurology, London, United Kingdom.
出版信息
Epilepsy Curr. 2023 May 19;23(4):245-250. doi: 10.1177/15357597231176234. eCollection 2023 Jul-Aug.
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.
摘要
近年来，用于测试癫痫基因疗法的临床前研究显著增加。其中一些疗法已进入临床试验阶段，并正在单基因或局灶性难治性癫痫患者中进行测试。本文概述了显示出临床转化潜力的临床前研究的现状。具体而言，我们关注那些在动物模型中已证明对癫痫发作有明显效果且有可能转化到临床环境中的基因疗法。我们讨论了针对病因的疗法和对症治疗的疗法。我们相信，未来几年对于确定基因疗法治疗癫痫患者的潜力至关重要。
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