Figure 5
From: Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy
Incomplete detoxification of ADA metabolites in thymocytes from PEG-ADA-treated mice. Tandem MS measurement of dAdo (A) (2 weeks, n = 10, 10; 3 month, n = 14, 14; 6 months, n = 10, 10; 9 months, n = 5, 5; 12 months, n = 6, 6) and Ado (B) (2 weeks, n = 9, 9; 3 month, n = 10, 14; 6 months, n = 8, 10; 9 months, n = 8, 8; 12 months, n = 5, 5) levels in unfractionated thymocyte populations from control (Ada+/+), untreated and PEG-ADA-treated Ada−/− mice at different time points. (C) Reversed phase HPLC measurement of dATP levels of unfractionated thymocyte populations from control (Ada+/+), untreated (2 weeks) and PEG-ADA-treated Ada−/− (6 months) mice (n = 12, 7, 5). Plots in (A-C) were generated by GraphPad Prism 7 software. (D) Tandem MS measurement of Ado and dAdo levels in FACS-sorted thymocyte populations from control (Ada+/+) and PEG-ADA-treated Ada−/− mice at 6-month-time point (n = 6, 8). Bars represent mean values. *P < 0.05, **P < 0.01 and ***P < 0.001. NS = statistically not significant.
