A single administration of a targeted gene therapy may offer long-lasting relief from muscle spasticity caused by spinal cord injury.
Gene Therapy Approach Shows Promise for Treating Spinal Spasticity
Researchers have evaluated a gene therapy strategy designed to restore the nerve signaling that controls muscle activity. The therapy targets two key components: the neurotransmitter GABA and the vesicular GABA transporter (VGAT).
The treatment was tested in a rat model of chronic spinal cord injury-induced spasticity. Animals showed a progressive reduction in spasticity, with significant improvements observed weeks after a single treatment.
How the Therapy Works
The therapy uses an inactive virus to deliver therapeutic genes directly into spinal cord cells located near the injury site. The goal is to restore normal function to spinal neurons involved in motor control. Results showed that the therapy:
- Partially restored normal spinal reflex function.
- Increased expression of therapeutic genes in spinal neurons critical for movement.
Safety and Longevity
Safety tests conducted in other animal models were encouraging. The treatment vehicles remained confined to the targeted spinal cord region, with no observed negative effects on motor or sensory function for up to 4.5 years.
Broader Implications
These findings suggest that a single administration of this gene therapy may provide a long-lasting reduction of muscle spasticity, not only from spinal cord injury but potentially for other neurological conditions as well.