Taken together, our results indicate that therapy through replacement of deficient ADAR2 protein is a logical approach to therapeutic intervention for ALS that could be effective in the majority of ALS patients and that given the demonstrated effects and safety of the clinical use of AAV, gene therapy using AAV9-hADAR2 is a promising therapeutic strategy for ALS. This evidence concerns the gene ADARB1 and amyotrophic lateral sclerosis.