AAV9-synapsin-caveolin-1 (AAV9-SynCav1) gene delivery preserves motor neuron and neuromuscular junction morphology, motor function and body weight, and extends survival in hSOD1 G93A mice.

AAV9-SynCav1 gene therapy significantly prolongs survival, preserves motor neurons and motor neuron morphology in the ventral horn of the spinal cord, and preservers NMJs in the tibialis muscle of hSOD1 mice. We are currently testing if alternative routes of AAV9-SynCav1 delivery (e.g., intrathecal, intraventricular, systemic) can recapitulate the neuroprotective effects achieved using subpial delivery.