Will Gene Therapy Be The Answer To Nerve Damage Problems?

Today's short post from sciencedaily.com/ (see link below) is a fascinating look at breakthroughs being made in the field of gene therapy for neuropathy (nerve damage). We're clearly at the beginning of new medical science when it comes to gene therapy and almost everything is still in the test stages. That's bad news for current neuropathy patients but the signs for the future are very encouraging indeed. This article looks at the research and breakthroughs being made when gene therapy is used to promote nerve regeneration. The current problem seems to be, that introducing new genetic structures in the body needs to be controllable and the genetic progress needs to be switched off when the goal is reached. Scientists are finding that the nerves do indeed regenerate but the process needs to be stopped at a given moment in time and currently they're not able to remove all vestiges of new active,genetic material. It may sound incredibly complex but this article explains it simply enough to be able to excite us at the prospects of restoring damaged nerves in the future. Hopefully, future generations will benefit but don't hold your breath that current patients will have easy access to this treatment - nothing in the research world works that quickly!

Gene therapy promotes nerve regeneration 
Date:January 18, 2019 Source:Netherlands Institute for Neuroscience - KNAW


Researchers from the Netherlands Institute for Neuroscience (NIN) and the Leiden University Medical Center (LUMC) have shown that treatment using gene therapy leads to a faster recovery after nerve damage. By combining a surgical repair procedure with gene therapy, the survival of nerve cells and regeneration of nerve fibers over a long distance was stimulated for the first time. The discovery, published in the journal Brain, is an important step towards the development of a new treatment for people with nerve damage.


During birth or following a traffic accident, nerves in the neck can be torn out of the spinal cord. As a result, these patients lose their arm function, and are unable to perform daily activities such as drinking a cup of coffee. Currently, surgical repair is the only available treatment for patients suffering this kind of nerve damage. "After surgery, nerve fibers have to bridge many centimeters before reaching the muscles and nerve cells from which new fibers need to regenerate are lost in large numbers. Most regenerating nerve fiber do not reach the muscles. The recovery of arm function is therefore disappointing and incomplete," explains researcher Ruben Eggers of the NIN.

Combination of treatments

By combining neurosurgical repair with gene therapy in rats, many of the dying nerve cells can be rescued and nerve fiber growth in the direction of the muscle can be stimulated.

In this study, the researchers used regulatable gene therapy with a growth factor that could be switched on and off by using a widely used antibiotic. "Because we were able to switch off the gene therapy when the growth factor was no longer needed, the regeneration of new nerve fibers towards the muscles was improved considerably," says Ruben Eggers.

A stealth gene switch

To overcome the problem of the immune system recognizing and removing the gene switch, the researchers developed a hidden version, a so-called 'stealth switch'. Professor Joost Verhaagen (NIN) explains: "The stealth gene switch is an important step forward towards the development of gene therapy for nerve damage. The use of a stealth switch improves the gene therapy rendering it even safer."

The gene therapy is not yet ready for use in patients. While the ability to switch off a therapeutic gene is a large step forward, the researchers still found small amounts of the active gene when the switch was turned off. Therefore, further research is needed to optimize this therapy.

The research was funded by Wings for Life, the International Spinal Research Trust and a donation from the Dwarslaesiefonds.


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Materials provided by Netherlands Institute for Neuroscience - KNAW. Note: Content may be edited for style and length.

https://www.sciencedaily.com/releases/2019/01/190118083157.htm