Fig. 7.

Glycosphingolipid correction by introduction of human GBA1. A: Human GBA1 was stably overexpressed in the gba1^−/− zebrafish background using the ubiquitin promoter. Zebrafish Gba1 and human GBA1 were visualized with ABP 1. B: Recombinant GBA1 was introduced by injection in the bloodstream of 2 dpf zebrafish with 10 or 36 × 10⁻⁶ U rGBA1 (Cerezyme®) and visualized with ABP 1. An equivalent of 36 × 10⁻⁶ U rGBA1 was labeled and used as control on gel. C: GlcSph levels were determined in picomoles per fish of uninjected control gba1^−/− zebrafish, gba1^−/− zebrafish stably overexpressing hGBA1, and gba1^−/− zebrafish infused with rGBA1. Data are depicted as mean ± SD and analyzed by one-way ANOVA (Dunnett’s test) with WT as control group. **P < 0.01 and ****P < 0.0001.