Lastly, in a phase 1/2 safety and efficacy clinical trial (NCT03952637), AAV9/GLB1 will be administered by intravenous infusion to subjects with type II GM1 gangliosidosis (ages 2–12 years) or patients aged 6 months to 1 year with a type I GM1 gangliosidosis. The gene discussed is GLB1; the disease is GM1 gangliosidosis.