Figure 5.
Reduction in GM2 ganglioside levels following intracranial injection of AAV9-Hex vectors. Four-month-old Tay-Sachs disease knock out mice received a single unilateral intracranial injection (left side) of either AAV9-GFP (109 vg) or a 2-vector mixture containing equal amounts (109 vg each) of AAV9-GFP and an AAV9-Hex vector, as designated on the left panel. Coronal sections of the brain were analyzed by immunohistochemistry (IHC) for green fluorescent protein (GFP) expression to indicate vector spread (a–d) and for GM2 ganglioside levels to assess the relative activity of Hex (e–h).