Because Ncald-ASOs applied by i.c.v. bolus injection efficiently knocked down NCALD levels in spinal cord of wild-type animals, we designed a double-blind pre-clinical study in SMA and HET mice, as shown in Figure 1B. In detail, at PND1 all animals received 30 μg SMN-ASOs by subcutaneous injection. The gene discussed is SMN2; the disease is proximal spinal muscular atrophy.