Blood, Vol. 110, Issue 8, 2855-2863, October 15, 2007

Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens
Blood Ide et al. 110: 2855

Supplemental materials for Ide et al, Vol. 110, Issue 8, 2855-2863.

Files in this Data Supplement:

• Figure S1. Hemophilia A mouse survival following intraperitoneal injection (JPG, 42.1 KB). -
  Mice injected with BU + CY on two consecutive days had significantly decreased survival versus mice injected with BU + CY or BU + ATS after a single 20U injection of hfVIII (P = .044, Gehan-Breslow test).
  
  
• Figure S2. Activated prothrombin times correlate with fVIII activity by chromogenic assay for treated and untreated mice (JPG, 37.4 KB). -
  Plasma was collected from transplanted E16^{−/−} mice 19 weeks following pfVIII-transduced HSCT as well as from control C57BL/6 and E16^{−/−} mice. Clot times were determined using 5µL of plasma in a modified APTT assay¹¹; all samples were clotted in duplicate with mean clot time plotted above. (A) Comparison of clot times from C57BL/6, naïve E16^{−/−}, and transplanted E16^{−/−} mice. A significant difference was observed between C57BL/6 and naïve E16^{−/−} mice (P = .001) and between naïve E16^{−/−} mice and transplanted E16^{−/−} mice (P −/− mice (39 seconds) was faster than the mean clot time for C57BL/6 mice (42 seconds), but no significant difference was found (P = .06). (B) Clot times are plotted versus fVIII activity levels by chromogenic assay. A significant correlation was found between these values (r² = 0.9, P
  
• Figure S3. T-cell depletion in peripheral blood and lymphoid organs following two doses of ATS (day-1, day 0) (JPG, 98.7 KB). -
  Representative flow diagrams are shown for mice killed on day +5. T cells were significantly depleted in the peripheral blood (P P = .089) or in the lymph nodes (P = .68).