Epilepsy Talk

Hope for the Future — Gene Therapy | October 7, 2016

Gene therapy is an experimental technique that uses genes to treat or prevent disease.

In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Researchers are testing several approaches to gene therapy, including:

  • 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.

Although gene therapy is a promising treatment option for a number of diseases (including inherited 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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  1. This is amazing!


    Comment by shamlin57 — October 7, 2016 @ 2:18 PM

  2. Pretty exciting stuff! 🙂


    Comment by Phylis Feiner Johnson — October 7, 2016 @ 4:15 PM

  3. Very cool! For the genetic condition I have that causes tumors to grow NF1 (Neurofibromatosis type 1) the 17th chromosome is where it is located. Last year, and again this year I have been able to hear a presentation about an ongoing study in large animals. For those of us who don’t know, this is second stage animal testing before it gets to human trials. In this study they spliced in the mutated gene in a sow so her offspring would have the genetic condition and be used then for testing and gathering data. (I know, mind-blowing!)

    I hope something along these lines can be used down the road for treatment in humans with epilepsy.

    Regarding the genetic condition I have, there are hundreds of mutations (if not more unknown) of the condition found in a few published studies. I arise this issue because each mutation is different and may require different treatment. The same would likely be true for epilepsy gene therapy.


    Comment by Travis — October 9, 2016 @ 12:43 PM

  4. Mind blowing, indeed. And I really appreciate your additional input. Thanks so much, Travis.

    P.S. There’s also cell transplantation to consider: Take a look at this link. (Not published yet.)



    Comment by Phylis Feiner Johnson — October 9, 2016 @ 1:05 PM

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    About the author

    Phylis Feiner Johnson

    Phylis Feiner Johnson

    I've been a professional copywriter for over 35 years. I also had epilepsy for decades. My mission is advocacy; to increase education, awareness and funding for epilepsy research. Together, we can make a huge difference. If not changing the world, at least helping each other, with wisdom, compassion and sharing.

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