Figure 6.

Reporter and therapeutic gene expression in brain of Parkin Q311(X)A mice after single i.t. injection of pre-transfected BMM. (A) Luc-transfected BMM (A, B) were injected through i.t. route (2.5 × 10⁶ cells/50 µL) to PD mice (black bars) or WT mice (striped bars). Mice were sacrifices 48 h after injection, perfused, brains were harvested and luciferase activity was measured in different brain regions [i.e. frontal cortex (1), midbrain (2), cerebellum (3), and pons (4)), and blood (5)]. Luciferase activity in each brain region was significantly greater in PD mouse brain regions than those in WT counterparts (A). Total brain/blood ratio of luciferase activity in PD mice was 25 times greater than in WT mice (B), indicating that BMM target inflamed brain tissues. (C) PD mice were injected with the same amount of GDNF-transfected BMM through i.t. route (black bars), or saline (white bars). Mice were sacrifices 48 h after injection, perfused, brains were harvested and GDNF expression was measured in different brain regions by ELISA. GDNF levels in each brain region were greater in PD mouse brain regions injected with GDNF-BMM than controls injected with saline. Negligible differences in GDNF levels in the blood were observed. Values are means ± SEM (N = 6). *p < 0.05 compared to WT healthy mice (A, B), or saline injected control mice (C). Individual data points shown in Supplementary Tables S8–S10.