SMN2 and spinal muscular atrophy: One notorious major breakthrough in biomedical research using transgenic mice carrying the human SMN2 gene led to the recent clinical approval of an AON, able to block an intronic splicing silencer in human SMN2 [71], increasing full-length SMN2 isoform expression, which compensates for the loss of SMN1 that causes spinal muscular atrophy [72,73,74,75].