Furthermore, 2 phase I/II clinical trials on safety and efficacy in GM1 gangliosidosis are ongoing, one to study a single-dose intravenous administration of AAV9/GLB1 (ClinicalTrials.gov NCT03952637) and the other to study a single-dose intra–cisterna magna administration of AAVhu68/GLB1 (ClinicalTrials.gov NCT04713475). The gene discussed is GLB1; the disease is GM1 gangliosidosis.