SOD1 and amyotrophic lateral sclerosis: After a single intrathecal injection of an AAVrh10 vector (4.2 × 1014 vector genomes) encoding miRNA targeting SOD1, the 22-year-old patient showed reduced post-mortem levels of SOD1 in spinal cord tissue and a transient improvement in right leg strength without change in vital capacity, whereas the 56-year-old patient who received immunosuppressive treatment had stable composite measure ALS functional scores and a stable vital capacity during 12 months.