SOD1 and amyotrophic lateral sclerosis: Among clinical trials, spinal ganglion toxicity was seen at autopsy in a patient with familial amyotrophic lateral sclerosis (ALS) and mutations in the gene encoding superoxide dismutase 1 (SOD1) after intrathecal infusion of 4.2 × 1014 vg AAVrh10 containing anti-SOD1 microRNA (AAV-miR-SOD1).