AAV Expression of a Blood-Brain Barrier-Penetrating Form of β-galactosidase Normalises GM1 Ganglioside Storage in Mice

Researchers assessed the therapeutic efficacy of a systemic infusion of an AAV vector carrying a gene expressing a blood-brain barrier-penetrable enzyme under the control of a liver-specific promotor in GM1 ganglioside (GM1) gangliosidosis model mice.