Moreover, the increased survival we obtained using AAV9-UBA1 in the severe “Taiwanese” SMA mouse model (with a life expectancy of ~9 days in our hands at present) is very similar in magnitude to the increase in survival previously reported in studies restoring SMN with AAV9 in comparable severe SMA mouse models (34) (in contrast to studies using the milder Δ7 SMA mouse model). The gene discussed is UBA1; the disease is proximal spinal muscular atrophy.