Figure 5.

CNS pathology is improved after AAV4βgluc delivery to the lateral ventricles of MPS VII mice. A-M, Lysosomal storage is observed throughout the brain in untreated (A, C, E, G, I, K, M) but reduced in treated (B, D, F, H, J, L, N) mutant mice. G-J, The hippocampus (G, H) and the striatum (J) are nearly devoid of remaining storage 4 weeks after gene transfer. A-N, Evaluation of the ependyma (A, B), meninges (C, D), cerebral cortex (E, F), cerebellum (K, L), and olfactory bulb (M, N) shows extensive improvements in lysosomal distention after AAV4βgluc delivery. Scale bar, 20 μm.