Biochemical analysis of tissue homogenates collected from transplanted MPS VII mice 1 year after engraftment and from untreated MPS VII controls.

Final values for enzyme-specific activity are expressed as average percent +/+ activity. (A) GUS activity showing increases in heart, liver, lung, and spleen and only modest increases in brain and kidney. (B) Decreased α-galactosidase activity was observed in tissues with the highest GUS activity, confirming lysosomal correction of GAG biochemistry. Levels of α-galactosidase activity remain high in brain and kidney, reflecting the low GUS activity seen in panel A. ▨ indicates untreated MPS VII; ■, 3x neonatal BMT.