There are no approved therapies for spinal cord injury. There are about 300,000 people living with chronicspinal cord injury in US. For the vast majority of them, the injury left them incapable of walking or, if theyhave suffered a cervical SCI, entirely dependent upon others for assistance in all of their activities of dailyliving from feeding to personal care. We are developing a neuromodulating gene therapy for treating bothacute and chronic SCI patients that will be delivered as a single injection to spinal cord.Neuromodulation after injury has the potential to restore functional recovery, as shown by a number ofdifferent investigators. After SCI there is an electrochemical imbalance that prevents motor neurons fromeliciting normal muscle actions that are critical for movement of arms and legs. This imbalance is partlycaused by a decrease in the chloride transporter called KCC2. It has been shown that by restoring normallevels of this transporter in spared spinal cord neurons, paralyzed mice regain walking ability. Furthermore,enhancement of KCC2 reduces pain and spasticity in rodent models. This grant application aims to examinea new therapy that has the potency and properties to be translated towards human clinical studies, bytesting them in preclinical models.
Public Health Relevance Statement: Narrative
Paralysis, neuropathic pain and spasticity are debilitating clinical consequences of spinal cord injury and there
are no approved therapies. We are developing a one-time-treatment that can be injected to cerebrospinal fluid.
By restoration of excitation/inhibition balance at spared spinal cord tissue, our therapy has the potential to
improve functional recovery, chronic pain and spasticity after acute and spinal cord injury.
Project Terms: 