Figure 4.

Disease-Associated Lesions in the Brain of MPS IIIA Mice at 25 Weeks Post-injection

Disease lesions associated with MPS IIIA were analyzed at 25 weeks post-treatment. (A and B) Quantification of the secondary accumulation of GM2 (A) and GM3 (B) ganglioside in brain slice 3. Male low dose-treated MPS IIIA mice #422, #383, and #448 were outliers, exhibiting no reduction in gangliosides (compare with MPS IIIA vehicle mice). This is in keeping with the low amount of SGSH activity and the high amount of HS recorded for these mouse samples (Figures 2 and 3) (C) Endosomal/lysosomal system expansion assessed by LIMP-2 staining in inferior colliculus. (D) Number of axonal spheroids, assessed by ubiquitin staining in inferior colliculus. (E) Extent of astrogliosis, assessed by GFAP staining in inferior colliculus. (F) Number of reactive amoeboid-shaped microglia, assessed by isolectin B4 staining, in dentate gyrus. *p < 0.05, ****p < 0.0001 calculated from one-way ANOVA with Bonferroni’s multiple comparisons test.