A study in Science Translational Medicine reports that intravenous AAV gene therapy extended lifespan and improved clinical outcomes in a feline model of Sandhoff disease. The therapy delivered the HEXA and HEXB enzymes linked to GM2 ganglioside breakdown, aiming to reduce lysosomal storage pathology. The work distinguishes itself by evaluating an intravenous delivery route, a strategy not previously tested in this disease context, where earlier approaches have focused on intrathalamic or cerebrospinal fluid administration. The authors report mitigation of pathological MRI changes in treated cohorts. For translational teams, the result is a practical reference point for delivery route feasibility in lysosomal storage diseases, where CNS access remains a major challenge. While preclinical, the dataset adds to the growing body of evidence that systemic dosing strategies may be able to support meaningful CNS exposure under certain vector and disease conditions.