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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

Arteries of the Lower Limbs

Arteries of the Lower Limbs

Epilepsy is a chronic neurological disease marked by recurrent, unpredictable seizures. These seizures are caused by abnormal electrical discharges in the brain, leading to behavior, sensation, or consciousness alterations. They can also cause transient impairment of awareness, interfering with daily activities.
Various factors can trigger epilepsy, including genetic factors, brain damage, metabolic causes, and unknown etiology. Diagnosis of epilepsy involves electroencephalography (EEG), which...

What is Genetic Engineering?

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Related Experiment Video

Updated: May 29, 2025

Electrophoretic Delivery of γ-aminobutyric Acid GABA into Epileptic Focus Prevents Seizures in Mice

Electrophoretic Delivery of γ-aminobutyric Acid GABA into Epileptic Focus Prevents Seizures in Mice

Published on: May 16, 2019

State-of-the-art gene therapy in epilepsy.

Matthew C Walker¹

¹Department of Clinical and Experimental Epilepsy, UCL Queen Square Institute of Neurology, University College London, London, UK.

Current Opinion in Neurology

|February 7, 2025

Summary

This summary is machine-generated.

Gene therapy is rapidly advancing for epilepsy treatment, offering targeted, permanent solutions. Recent developments include antisense oligonucleotides and vector-based gene therapies, with future approaches focusing on precise network excitability regulation.

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Last Updated: May 29, 2025

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Preparation and Implantation of Electrodes for Electrically Kindling VGAT-Cre Mice to Generate a Model for Temporal Lobe Epilepsy

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Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats

Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats

Published on: May 16, 2025

Area of Science:

Neuroscience
Genetics
Molecular Biology

Background:

Epilepsy genetics and mechanisms have seen significant advancements.
Gene therapy tools, particularly viral vectors, have improved considerably.
Epilepsy is an ideal target for gene therapy due to its genetic underpinnings.

Purpose of the Study:

To review recent developments in gene therapy for epilepsy.
To highlight advances in gene therapy tools and their application to epilepsy treatment.

Main Methods:

Review of recent literature on gene therapy for epilepsy.
Focus on antisense oligonucleotide therapies and vector gene therapy.
Exploration of emerging techniques like optogenetics and DREADDs.

Main Results:

Antisense oligonucleotide therapies are progressing towards clinical trials for monogenic epilepsies.
Vector gene therapy offers permanent, one-off treatment by delivering genes directly to the brain.
Promising approaches include NPY/Y2 receptor expression, GluK5 knockdown, and Kv1.1 overexpression for focal epilepsy.

Conclusions:

Gene therapy presents a novel treatment for all epilepsy types, not just genetic forms.
It may offer a precise, permanent alternative to current drug and surgical treatments.
Future gene therapies aim for targeted treatment with potentially fewer adverse effects.