SOD1 and amyotrophic lateral sclerosis: The success of gene-targeting therapy in spinal muscular atrophy has raised expectations of the prospect of similar strategies in other neurological disorders, including ALS.21 Phase 1 and 2 antisense oligonucleotide trials targeting wild-type SOD1 have been completed22 and several phase 1/2 studies are underway for C9ORF72 hexanucleotide repeat expansion-associated ALS.