Fig. (2).
Design of in vivo transplant studies and primary transplant results. A) Study design. Lin- bone marrow cells from β-thalassemia mice (Hbbth1/th1 ; β-thal CD45.2) mice were transduced with lentiviral vectors and infused in 58 syngenic recipients. Four months after primary transplantation, the remaining 54 animals were sacrificed and bone marrow cells were injected in 108 secondary normal mice. The numbers of prematurely dead mice are indicated on top of the arrows. B) Mean (± SD) percentage chimerism estimated in primary transplant recipients. C) Mean VCN (± SD) pre-transplantation (in vitro), and in blood at 2 and 3 months after transplantation (2M, 3M), and in bone marrow after sacrifice (whole bone marrow and after culture for CFCs). D) Proportion of therapeutic β-globin chains and correction of the β-thalassemic phenotype (hemoglobin concentration and reticulocyte percentage) per group of mice. Horizontal bar represents the mean.