Intrathecal delivery of AAVs containing an artificial miRNA suppressing superoxide dismutase 1 (SOD1) (a genetic cause of familial amyotrophic lateral sclerosis (ALS)), was shown to be efficient in SOD1 gene silencing in nonhuman primates, yet it did induce peripheral immune responses [201]. This evidence concerns the gene SOD1 and familial amyotrophic lateral sclerosis.