However, findings of toxicity of overexpressed SMN in the mouse model should be interpreted with caution because a different promoter was used in the AAV9 vector to express SMN. Furthermore, unlike the intrathecal route of delivery in the mouse study, SMA patients receive onasemnogene through intravenous administration (Al-Zaidy et al., 2019; Mercuri et al., 2021; Weiß et al., 2022). The gene discussed is SMN2; the disease is proximal spinal muscular atrophy.