296151.fig.003a
(a)
296151.fig.003b
(b)
Figure 3: Enzyme replacement therapy with THLs in a mouse model of type VII mucopolysaccharidosis. (a) GUSB enzyme activity in GUSB null (−) fibroblasts and in fibroblasts obtained from wild type (+) mice. Fibroblasts were treated either with saline or with TfRMAb-targeted THLs encapsulated with the pCMV-GUSB expression plasmid. Data are mean ± SE ( ), and statistical significance was determined with ANOVA and Dunnett’s test. The difference in GUSB enzyme activity in the THL-treated cells is significantly different from the untreated cells from the GUSB null mice ( ). (b) GUSB enzyme activity in brain and five other organs of GUSB null mice removed at 48 h after single intravenous administration of either saline or 10 ug/mouse of pCMV-GUSB plasmid DNA encapsulated in TfRMAb-targeted THLs. Mean ± SE ( = 4-5 mice/group). The difference in GUSB enzyme activity in the THL-treated mice, as compared to the saline-treated mice, is significant ( ), in all organs, except the heart. From [36].