News Finding: Enzyme expression in liver cells reached levels greater than 200-fold higher than in wild-type mice.
Paper Finding: All ZFN+donor groups exhibited dose-dependent IDS activity levels in the liver at 1 month (mean values of 265, 1,756, and 2,479 nmol/ hr/mg for the low-, mid-, and high-dose groups, respectively; Figure 3A) and 4 months (519, 1,696, and 5,355 nmol/hr/mg protein for the low-, mid-, and high-dose groups, respectively; Figure 3B ) post-treatment.
News Context: Treated mice, at both one and four-months after injection, exhibited dose-dependent expression of IDS enzyme in blood and peripheral tissues, including the spleen, kidney, lung, heart, and skeletal muscle. Enzyme expression in liver cells reached levels greater than 200-fold higher than in wild-type mice. In all tested peripheral organs, treatment at the highest dose resulted in greater than 95% reduction in glycosaminoglycan (GAG) substrate levels.
Paper Context: Detection of hIDS protein thus required the presence of both ZFNs and the hIDS donor. All ZFN+donor groups exhibited dose-dependent IDS activity levels in the liver at 1 month (mean values of 265, 1,756, and 2,479 nmol/ hr/mg for the low-, mid-, and high-dose groups, respectively; Figure 3A) and 4 months (519, 1,696, and 5,355 nmol/hr/mg protein for the low-, mid-, and high-dose groups, respectively; Figure 3B ) post-treatment. The levels of IDS activity in all three ZFN+donor groups were significantly higher than control MPS II mice and were 20-, 66-, and 207-fold higher than levels found in wild-type mice at 4 months ( Figure 3B ).