SynCav1 gene therapy via subpial delivery is beneficial in familial ALS model
Aug. 22, 2022
Neuron-targeting of caveolin-1 (Cav-1) using AAV9 delivery and a synapsin-driven Cav-1 engineered construct, SynCav1, showed therapeutic potential in the hSOD1G93A model of familial amyotrophic lateral sclerosis (ALS).