Gene Therapy for Spinal Cord Injury Neuropathy

In a new study published in the journal Molecular Therapy, researchers from an international team, led by scientists at the University of California San Diego School of Medicine have found that gene therapy that inhibits targeted nerve cell signaling effectively reduces neuropathic pain in mice with spinal cord or peripheral nerve injuries. 

Given the current U.S. population size of 324 million people, a recent estimate showed that the annual incidence of spinal cord injury (SCI) is approximately 54 cases per one million people in the United States, or about 17,500 new SCI cases each year. New SCI cases do not include those who die at the scene of the accident.

There is currently no effective therapy for neuropathy or spinal cord injury. Pharmacological therapies come with many undesirable side effects. Gene therapy has proven to be an attractive possibility. 

Study Development and Results 

For the study, the team injected an adeno-associated virus carrying a pair of transgenes that encode gamma-aminobutyric acid (GABA) into mice with sciatic nerve injuries and consequential neuropathic pain. GABA is an inhibitory neurotransmitter that blocks impulses in nerve cells, including pain signals. 

The delivery and expression of these transgenes (GAD65 and VGAT) were targeted to the area of sciatic nerve injury and there were no side effects. The transgenes lead to the production of GABA, which inhibits pain-signaling neurons in mice for up to 2.5 months. 

According to the study, no side effects were seen between 2-13 months post-treatment in naive adult mice, pigs, and non-human primates. 

This approach could be used to develop new treatments for patients suffering from spinal cord or peripheral nerve injury-induced neuropathic pain. 


Scott LaFee. (2022, May 9). Gene Therapy Shows Promise in Treating Neuropathy from Spinal Cord Injuries. UC San Diego News Center, UC San Diego. Retrieved from:

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