Since every SMA patient has at least one functioning SMN2 gene, it has become a target for therapeutic interventions, and most pre-clinical studies have focused on up-regulating SMN levels by some means [14], [15], [16], [17], [18], [19], [20], [21], [22], [23], [24]. The gene discussed is SMN2; the disease is proximal spinal muscular atrophy.