Figure 2
Transient fVIII activity and subsequent inhibitor formation following BU + CY conditioning and HSCT/ BDDpfVIII gene therapy. Mice were conditioned with 35 mg/kg BU and 200 mg/kg CY on days − 3 and − 2, then received a transplant of 3 × 105 BDDpfVIII-transduced sca-1+ cells on day 0. (A) FVIII activity levels on days + 7 and + 14 as measured by chromogenic fVIII assay. Dashed line indicates lower limit of detection. Mice were assayed biweekly after day + 14 and no fVIII activity was detected in any mice. (B) Anti-BDDpfVIII inhibitory antibody titers as determined by the Bethesda assay. Solid line indicates upper limit of detection.
