Gene therapy promotes nerve regeneration

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

During delivery or as a result of a road accident, the nerves in the neck can be pulled from the spinal cord. As a result, patients lose the function of their arm and are unable to perform daily activities such as drinking a cup of coffee. Currently, surgical repair is the only treatment available for patients suffering from this type of nerve damage. "After the surgery, the nerve fibers must cross several centimeters before reaching the muscles and the nerve cells from which new fibers must regenerate are lost." Most regenerating nerve fibers do not reach the muscles. The recovery of arm function is therefore disappointing incomplete, "says NIN researcher Ruben Eggers.

Combination of treatments

By combining neurosurgical repair with gene therapy in the rat, many dying nerve cells can be saved and nerve fiber growth directed to the muscle can be stimulated.

In this study, researchers used a regulatable gene therapy with a growth factor that can be activated and deactivated using a widely used antibiotic. "Because we were able to turn off gene therapy when the growth factor was no longer needed, the regeneration of new nerve fibers to the muscles has improved dramatically," Ruben Eggers says.

A stealth gene switch

To overcome the problem of the recognition and elimination of the genetic switch by the immune system, the researchers developed a hidden version, called a "stealth switch". Professor Joost Verhaagen (NIN) explains: "The stealth gene change is an important step forward in the development of gene therapy for nerve damage." The use of a stealth switch improves gene therapy by making it even safer. "

Gene therapy is not yet ready for use in patients. Although the possibility of disabling a therapeutic gene is a big 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.

Provided by
Dutch Institute of Neuroscience – KNAW