Results of in vivo studies in a mouse model of Gaucher disease showed that intravenous administration of AAV9 carrying the mouse GBA1 gene controlled by human cytomegalovirus enhancer and promoter (3 × 1011 vg/animal) restores the activity of GBA1 in many organs, prolongs lifespan, and improves neuropathological changes in mice [130]. This evidence concerns the gene GBA1 and Gaucher disease.