Previous studies in murine and feline models of GM1 gangliosidosis demonstrated that adeno-associated viral (AAV) vectors can be used to deliver the normal GLB1 coding sequence to neurons, resulting in long-term expression of the β-gal enzyme.4,5 β-gal is secreted by transduced cells and can be taken up by surrounding cells via binding to the mannose-6-phosphate receptor, resulting in widespread distribution of the enzyme.5,6 This phenomenon allows for the correction of storage lesions throughout the CNS after a small number of vector injections into the brain parenchyma. Here, GLB1 is linked to GM1 gangliosidosis.