SOD1 and amyotrophic lateral sclerosis: Mutations in the superoxide dismutase 1 (SOD1) gene account for approximately 15% of familial ALS cases.564 Designing ASO drugs that specifically target these SOD1 gene mutations and reduce the levels of the harmful SOD1 protein represents a promising therapeutic approach.561 ASO drugs targeting SOD1 have been used to treat familial ALS in human clinical trials and slow disease progression.561,747 This targeted gene selection makes the treatment more focused, significantly improving patient outcomes.