Figure 1.

Sulfamidase-Specific Activity in the CNS of Pigs Injected with AAV9 Vectors Carrying Different SGSH Expression Cassettes

(A) WT pigs at P60 were injected via cisterna magna with 4.5 × 10¹² GCs/kg of AAV9 encoding different human SGSH expression cassettes under the CMV promoter: WT SGSH, IDSspSGSH, SGSH-IRES-SUMF1, and IDSspSGSH-IRES-SUMF1. At 1 month after injection, sulfamidase-specific activity (nmol/17 h/mg of protein) was measured in the indicated 20 different areas of the CNS (F. cortex and O. cortex mean frontal and occipital cortex, respectively) and compared with control WT pigs treated with PBS. n = 5 animals for each group. Data represent mean ± SEM. (B) IHC staining with anti-human sulfamidase in main representative CNS areas of injected WT pigs. Scale bars: 300 μm.