Gene Therapy Restores Mobility in Spinal Cord Injuries, According to .NeuroRestore Scientists
Scientists at .NeuroRestore, a research institute in Switzerland, have successfully developed a groundbreaking gene therapy that stimulates nerve regrowth in spinal cord injuries, leading to restored mobility. The therapy was tested and proven effective in mice, showing promising results for potential human applications.
Typically, when the spinal cords of mice and humans are partially damaged, there is a natural repair process that allows for the recovery of motor function. However, in cases of complete spinal cord injuries, this repair process does not occur, resulting in permanent paralysis. Until now, there have been no successful strategies to restore motor function in cases of severe injuries.
Five years ago, researchers at .NeuroRestore demonstrated that nerve fibers can be regenerated across complete spinal cord injuries. However, they discovered that this alone was not enough to restore motor function, as the new fibers failed to connect to the correct locations on the other side of the injury. This missing connection hindered the restoration of mobility.
To address this issue, scientists collaborated with researchers from the University of California, Los Angeles (UCLA) and Harvard Medical School. Using advanced equipment at EPFL’s Campus Biotech facilities in Geneva, they conducted in-depth analyses to identify the specific type of neurons involved in spinal cord repair after partial injuries. They found that the regenerating axons needed to reconnect with their natural targets to restore motor function.
Based on these findings, the scientists developed a multipronged gene therapy. They activated growth programs in the identified neurons of mice, enabling them to regenerate nerve fibers. Additionally, they upregulated specific proteins to support the growth of these neurons through the injury site and administered guidance molecules to attract the regenerating nerve fibers to their natural targets.
Remarkably, mice with complete spinal cord injuries who received the gene therapy regained the ability to walk. Their gait patterns resembled those of mice with partial injuries who naturally resumed walking. This discovery revealed a crucial condition for successful regenerative therapies in restoring motor function after neurotrauma.
The researchers believe that a complete solution for treating spinal cord injuries will require a combination of approaches. In addition to gene therapy, they emphasize the importance of electrical stimulation of the spinal cord to maximize the potential of nerve fibers and the spinal cord to produce movement.
While there are still obstacles to overcome before this gene therapy can be applied to humans, the scientists see this development as a significant step towards treating spinal cord injuries effectively. They are optimistic about the potential impact of their research in the years to come.
The study, titled “Recovery of walking after paralysis by regenerating characterized neurons to their natural target region,” was published in the journal Science on September 22, 2023. The authors of the study include Jordan W. Squair, Marco Milano, Alexandra de Coucy, Matthieu Gautier, Michael A. Skinnider, Nicholas D. James, Newton Cho, Anna Lasne, Claudia Kathe, Thomas H. Hutson, Steven Ceto, Laetitia Baud, Katia Galan, Viviana Aureli, Achilleas Laskaratos, Quentin Barraud, Timothy J. Deming, Richie E. Kohman, Bernard L. Schneider, Zhigang He, Jocelyne Bloch, Michael V. Sofroniew, Gregoire Courtine, and Mark A. Anderson.
While further development and testing are necessary, this breakthrough gene therapy brings hope for the future of treating spinal cord injuries and restoring mobility to those affected.