Gene Therapy for Complete Spinal Cord Injuries

Published 2023-09-27

Researchers from the NeuroX Institute at the Swiss Federal School of Technology recently published research results in the journal Science, pertaining to the successful application of gene therapy in mice with complete spinal cord injuries. Contrary to previous partial restoration of neurological function after complete spinal cord injury, this study is the first to demonstrate full recovery of motor function. Using single-cell nuclear RNA sequencing, the team identified the specific axons that need to be repaired to restore motor function. This approach was based on the identification of neuronal subpopulations that restore walking after incomplete SCI. The resulting gene therapy product upregulates certain proteins that help the neurons grow through the damaged tissue, and added molecules that guide those regenerating nerves to their targets on the other side. The mice treated, recovered full motor function 8 weeks after a complete crush injury to the spinal cord.