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.”

 

To subscribe to Epilepsy Talk and receive the latest articles by email, simply go to the bottom of the right column and click on “Follow”

 

Resources:

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

http://wesscholar.wesleyan.edu/cgi/viewcontent.cgi?article=1127&context=div3facpubs

http://www.ninds.nih.gov/news_and_events/news_articles/news_article_epilepsy_gene_therapy.htm

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

 

---