For example, researchers have designed specific ASOs to reduce the expression of detrimental SMN2 isoforms while facilitating therapeutic SMN2 isoforms to inhibit spinal muscular atrophy, leading to the first splice-switching therapy (Spinraza) approved by FDA in 2016.14 Besides, a variety of preclinical studies and clinical trials have confirmed the great promise of ASOs in the treatment of cancer. This evidence concerns the gene SMN2 and spinal muscular atrophy.