Figure 4.

Reduction in gag storage. MPS IIIA (a) or MPS I (b) skin fibroblasts were transfected with shEXTL2.1, shEXTL2.2, shEXTL2.3 or shEXTL3.2. A mock transfection consisting of lipofectamine 2000 reagent only served as control. After 48 h, cells were harvested and the lysosomal fraction recovered after Percoll gradient centrifugation. Results are the mean±standard deviation of two experiments performed in duplicate and are expressed as the percentage of the mock control. ^*Significant difference from mock transfection, P<0.05 (ANOVA, Tukey's HSD).