Epilepsy Talk

Hope for the Future — Gene Therapy | October 26, 2025

Imagine if you had genetic intractable epilepsy and you were able to have it treated by inserting a simple gene into your cells.

Without surgery. Without drugs.

Sound scary? Too futuristic?

Well, that research is here. Now. And it’s called “gene therapy.”

Among the approaches to gene therapy, tests are being conducted on:

• Replacing a mutated gene that causes disease with a healthy copy of the gene.
• Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
• Introducing a new gene into the body to help fight a disease.

But, although gene therapy is a promising treatment option for a number of diseases (including epilepsy, inherited genetic disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective.

Gene therapy is currently only being tested for the treatment of diseases that have no other cures.

Viruses can also be used to introduce genes for proteins such as GABA into non-neuronal cells.

These cells are then transplanted into the brain to act as “factories” to produce potentially therapeutic proteins.

One advantage of gene therapy is that it can alter the cells in just one part of the brain.

Researchers can control the activity of the introduced genes by using a genetic “switch” that responds to antibiotics or other chemicals.

This allows doctors to turn the gene therapy off if it causes intolerable side-effects or other problems.

Theoretically, this type of therapy should last longer and cause fewer side-effects than medication. 

Also, researchers are working to identify more gene variations and to understand how they influence individual responses to treatment.

Eventually, it may become possible to test for these genetic variations and, to use the information to prescribe more effective treatments.

Researchers also may be able to develop ways of overcoming genetic resistance to treatment.

“What effect a compound is going to have partly depends on where in the seizure circuit that new compound or gene is being placed.

You could put the same chemical in two places and get two different results,” said Dr. Edward Bertram III, a professor of neurology at the University of Virginia.

“That is going to be the issue as they try to develop this: Where should we be putting this to have the best effect?

On the promising side, they put (the gene) in a restricted area and had an effect. That is a great first step.”

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Resources:

http://www.sciencedaily.com/releases/2010/08/100825093541.htm.

https://www.ucl.ac.uk/news/2023/dec/new-gene-therapy-could-significantly-reduce-seizures-severe-childhood-epilepsy

https://ghr.nlm.nih.gov/primer/therapy/genetherapy

https://pmc.ncbi.nlm.nih.gov/articles/PMC9132249/

https://journals.lww.com/co-neurology/fulltext/2025/04000/state_of_the_art_gene_therapy_in_epilepsy.5.aspx

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