Recently, longer-lived intermediate models were generated by administration of sub-optimal doses of exon 7 inclusion-promoting ISS-N1 antisense oligonucleotides (ASOs, Box 1) to human SMN2-carrying SMA mice (to promote an insufficient increase of FL-SMN for complete rescue) (Hosseinibarkooie et al., 2016; Zhou et al., 2015). Here, SMN2 is linked to proximal spinal muscular atrophy.