Gene therapy for epilepsy : An emerging, promising approach for a serious neurological disorder
(2026) In Journal of Internal Medicine 299(3). p.302-327- Abstract
Gene therapy is emerging as a groundbreaking strategy for treating epilepsy, offering new hope to patients who do not respond to conventional medications. Despite advancements in anti-seizure treatments, nearly 30%–40% of individuals with epilepsy continue to experience uncontrolled seizures, highlighting the urgent need for more effective and long-lasting solutions. By addressing the underlying causes of epilepsy at the genetic level, gene therapy represents a paradigm shift in treatment. Two key approaches are being explored: (1) activating or supplementing beneficial genes to suppress seizures and (2) silencing harmful genes or pathways that contribute to epilepsy. To achieve these objectives, viral vectors, such as adeno-associated... (More)
Gene therapy is emerging as a groundbreaking strategy for treating epilepsy, offering new hope to patients who do not respond to conventional medications. Despite advancements in anti-seizure treatments, nearly 30%–40% of individuals with epilepsy continue to experience uncontrolled seizures, highlighting the urgent need for more effective and long-lasting solutions. By addressing the underlying causes of epilepsy at the genetic level, gene therapy represents a paradigm shift in treatment. Two key approaches are being explored: (1) activating or supplementing beneficial genes to suppress seizures and (2) silencing harmful genes or pathways that contribute to epilepsy. To achieve these objectives, viral vectors, such as adeno-associated viruses and lentiviruses, have shown promise in delivering targeted genetic interventions. In parallel, cutting-edge techniques such as optogenetics, chemogenetics, and clustered regularly interspaced short palindromic repeat-based gene editing are enhancing the precision of these therapies, enabling greater control over neuronal activity. However, significant challenges exist, including ensuring safe and efficient gene delivery, maintaining long-term therapeutic effects, and mitigating potential side effects. This review examines recent developments in gene therapy for epilepsy, assessing its potential to deliver targeted, long-lasting treatments for drug-resistant epilepsy. By examining current strategies, therapeutic targets, and emerging technologies, it provides insights into the promising future of gene therapy as a transformative tool in epilepsy treatment and summarizes current clinical trials utilizing gene and cell therapy technologies for epilepsy.
(Less)
- author
- Ledri, Marco LU and Kokaia, Merab LU
- organization
- publishing date
- 2026-03
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- chemogenetics, epilepsy, gene therapy, optogenetics, targeted therapy, viral vectors
- in
- Journal of Internal Medicine
- volume
- 299
- issue
- 3
- pages
- 26 pages
- publisher
- Wiley-Blackwell
- external identifiers
-
- pmid:41366856
- scopus:105024685748
- ISSN
- 0954-6820
- DOI
- 10.1111/joim.70059
- language
- English
- LU publication?
- yes
- additional info
- Publisher Copyright: © 2025 The Author(s). Journal of Internal Medicine published by John Wiley & Sons Ltd on behalf of Association for Publication of The Journal of Internal Medicine.
- id
- cedb1d94-9bb7-46c7-bec2-0447952fa338
- date added to LUP
- 2026-03-02 15:22:32
- date last changed
- 2026-03-02 15:23:18
@article{cedb1d94-9bb7-46c7-bec2-0447952fa338,
abstract = {{<p>Gene therapy is emerging as a groundbreaking strategy for treating epilepsy, offering new hope to patients who do not respond to conventional medications. Despite advancements in anti-seizure treatments, nearly 30%–40% of individuals with epilepsy continue to experience uncontrolled seizures, highlighting the urgent need for more effective and long-lasting solutions. By addressing the underlying causes of epilepsy at the genetic level, gene therapy represents a paradigm shift in treatment. Two key approaches are being explored: (1) activating or supplementing beneficial genes to suppress seizures and (2) silencing harmful genes or pathways that contribute to epilepsy. To achieve these objectives, viral vectors, such as adeno-associated viruses and lentiviruses, have shown promise in delivering targeted genetic interventions. In parallel, cutting-edge techniques such as optogenetics, chemogenetics, and clustered regularly interspaced short palindromic repeat-based gene editing are enhancing the precision of these therapies, enabling greater control over neuronal activity. However, significant challenges exist, including ensuring safe and efficient gene delivery, maintaining long-term therapeutic effects, and mitigating potential side effects. This review examines recent developments in gene therapy for epilepsy, assessing its potential to deliver targeted, long-lasting treatments for drug-resistant epilepsy. By examining current strategies, therapeutic targets, and emerging technologies, it provides insights into the promising future of gene therapy as a transformative tool in epilepsy treatment and summarizes current clinical trials utilizing gene and cell therapy technologies for epilepsy.</p>}},
author = {{Ledri, Marco and Kokaia, Merab}},
issn = {{0954-6820}},
keywords = {{chemogenetics; epilepsy; gene therapy; optogenetics; targeted therapy; viral vectors}},
language = {{eng}},
number = {{3}},
pages = {{302--327}},
publisher = {{Wiley-Blackwell}},
series = {{Journal of Internal Medicine}},
title = {{Gene therapy for epilepsy : An emerging, promising approach for a serious neurological disorder}},
url = {{http://dx.doi.org/10.1111/joim.70059}},
doi = {{10.1111/joim.70059}},
volume = {{299}},
year = {{2026}},
}