Our strategy for disrupting the SMN2-ISSs in human SMA iPSCs was basedon three sgRNAs (sgRNA1, 2 and 3) that we designed to target ISS-N1 and one sgRNA (sgRNA4)to target ISS + 100 (Fig. 1A and Supplementary Fig.1A). The gene discussed is SMN2; the disease is proximal spinal muscular atrophy.