Firstly, they can be delivered to the brain relatively efficiently, for example, intrathecal delivery of ASO that modulates the splicing of SMN2 RNA for ventral spinal motor neurons have been successfully developed to treat spinal muscular atrophy (SMA).23 Furthermore, ASO-based therapies enable multi-targeting and can be adjusted sequentially to the disease stage.20 ASOs targeting mRNA or miRNA can be used not only to reduce expression, but also enhance it, thereby permitting the removal of malfunctioning proteins or the restoration of those with proper function. The gene discussed is SMN2; the disease is proximal spinal muscular atrophy.