News Finding: Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
  
  $SGMO $NTLA $EDIT $CRSP #genomeediting @username @username @username @username #mucopolysaccharidosis #MPSII #huntersyndrome
  
   
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: Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
  
  $SGMO $NTLA $EDIT $CRSP #genomeediting @username @username @username @username #mucopolysaccharidosis #MPSII #huntersyndrome
  
   
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 ).