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. 2017 Jun 6;6:43–53. doi: 10.1016/j.omtm.2017.05.009

Figure 2.

Figure 2

Uptake and Biodistribution of BMN 250 in Naglu−/− Mouse Brain

Naglu−/− mice were administered 100 μg ICV doses of BMN 250 or vehicle (controls) on study days 1, 5, 10, and 14. n = 6–8 animals per treatment group, except n = 2 for the naive Naglu+/− group. (A) NAGLU enzyme activity (mean ± SEM) measured in brain homogenate 1 or 28 days after the last ICV infusion. VEH, vehicle. ***p < 0.001, ****p < 0.0001. (B) Immunohistochemical signal of NAGLU (green) with DAPI nuclear stain (blue) in the septal, mid-hippocampal, and midbrain sections, showing bilateral biodistribution across the neuro-axis 1 day after the last ICV dose. (C) Representative high-resolution confocal images of NAGLU (green) co-stained with NeuN, GFAP, IBA1, and CD31 (red), showing localization of the NAGLU signal to neurons, astrocytes, microglia, and endothelia, respectively, 1 day after the last ICV infusion. (D) Percent of total NAGLU signal associated with each cell type, determined by quantitative analysis on co-stained sections 1 day after the last ICV infusion. (E) Representative high-resolution confocal images of NAGLU (green) co-immunostained with LAMP2 (red), showing localization of the NAGLU signal to lysosomes (yellow), indicated by arrows, 1 day after the last ICV infusion.