Studies on SMA type I patient cells and severe SMA mouse models demonstrated the efficacy of 8-mer ASO binding to GCRS, which not only elevated SMN levels but also increased the levels of Gemin 2 and Gemin 8 factors involved in snRNP biogenesis and Tra2-β1 and hnRNP Q, responsible for proper RNA splicing [63,64]. The gene discussed is SMN2; the disease is spinal muscular atrophy, type 1.