Figure 7.
Analysis of secondary F8−/−recipients of Lin−cells from in vivo transduced hCD46+/+F8−/−mice. (A) Bone marrow Lin− cells pooled from 4 CD46+/+/F8−/− mice at week 16 after in vivo transduction (Figure 6) were transplanted into 1050 Rad irradiated F8−/− hemophilia A mice. (B) Engraftment based on the percentage of hCD46-positive peripheral blood mononuclear cells (PBMCs). (C) ET3 activity measured in plasma by Coamatic FVIII assay. Activities of 100% and 5% are indicated by dotted lines. (D) Bleeding after tail clipping analyzed at week 10 after transplantation. For comparison, “healthy” CD46+/+ and hemophilia A CD46+/+/F8−/− mice are shown. Secondary recipients were kept alive without side effects for 10 weeks.