Researchers involving the DQBM Polymenidou Lab tested a gene therapy designed to restore inhibitory signalling in selected spinal cord segments. A single subpial injection of AAV9 vectors encoding GAD65 and VGAT significantly reduced spasticity in rats with chronic spinal cord injury.
The treatment increased inhibitory GABA-related activity within excitatory spinal neurons while preserving normal forelimb function. Safety studies in pigs and non-human primates found no neurological impairment or spinal toxicity, including 4.5 years after treatment in primates. Minimal vector material reached peripheral organs. The findings support further development, although clinical studies are required to establish safety and efficacy in patients.
DOI: https://doi.org/10.1016/j.ymthe.2026.06.008