» Articles » PMID: 37662470

Are Genetic Therapies for Epilepsy Ready for the Clinic?

Overview
Journal Epilepsy Curr
Publisher Sage Publications
Date 2023 Sep 4
PMID 37662470
Authors
Affiliations
Soon will be listed here.
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.

Citing Articles

Genetic Testing in Epilepsy: Improving Outcomes and Informing Gaps in Research.

Habela C, Schatz K, Kelley S Epilepsy Curr. 2024; :15357597241232881.

PMID: 39554273 PMC: 11562134. DOI: 10.1177/15357597241232881.


Recent advances and current status of gene therapy for epilepsy.

Cai A, Gao K, Zhang F, Jiang Y World J Pediatr. 2024; 20(11):1115-1137.

PMID: 39395088 DOI: 10.1007/s12519-024-00843-w.


Flavonoids as therapeutic agents for epilepsy: unveiling anti-inflammatory and antioxidant pathways for novel treatments.

Zhang Y, Hu X, Zou L Front Pharmacol. 2024; 15:1457284.

PMID: 39329119 PMC: 11424894. DOI: 10.3389/fphar.2024.1457284.


NORSE/FIRES: how can we advance our understanding of this devastating condition?.

Champsas D, Zhang X, Rosch R, Ioannidou E, Gilmour K, Cooray G Front Neurol. 2024; 15:1426051.

PMID: 39175762 PMC: 11338801. DOI: 10.3389/fneur.2024.1426051.


Neuroprotective Efficacy and Complementary Treatment with Medicinal Herbs: A Comprehensive Review of Recent Therapeutic Approaches in Epilepsy Management.

Anand A, Shrivastava A, Singh K, Barik R, Gayakwad D, S J CNS Neurol Disord Drug Targets. 2024; 24(1):60-73.

PMID: 39069797 DOI: 10.2174/0118715273332140240724093837.


References
1.
Ahonen S, Nitschke S, Grossman T, Kordasiewicz H, Wang P, Zhao X . Gys1 antisense therapy rescues neuropathological bases of murine Lafora disease. Brain. 2021; 144(10):2985-2993. PMC: 8634080. DOI: 10.1093/brain/awab194. View

2.
Nabbout R, Chemaly N, Chipaux M, Barcia G, Bouis C, Dubouch C . Encephalopathy in children with Dravet syndrome is not a pure consequence of epilepsy. Orphanet J Rare Dis. 2013; 8:176. PMC: 4225757. DOI: 10.1186/1750-1172-8-176. View

3.
Wykes R, Heeroma J, Mantoan L, Zheng K, MacDonald D, Deisseroth K . Optogenetic and potassium channel gene therapy in a rodent model of focal neocortical epilepsy. Sci Transl Med. 2012; 4(161):161ra152. PMC: 3605784. DOI: 10.1126/scitranslmed.3004190. View

4.
Colasante G, Qiu Y, Massimino L, Di Berardino C, Cornford J, Snowball A . In vivo CRISPRa decreases seizures and rescues cognitive deficits in a rodent model of epilepsy. Brain. 2020; 143(3):891-905. PMC: 7089667. DOI: 10.1093/brain/awaa045. View

5.
Burbano L, Li M, Jancovski N, Jafar-Nejad P, Richards K, Sedo A . Antisense oligonucleotide therapy for KCNT1 encephalopathy. JCI Insight. 2022; 7(23). PMC: 9746904. DOI: 10.1172/jci.insight.146090. View