Cell therapy for hemophilia

摘要

In this issue of Blood, Follenzi and colleagues demonstrate that transplantation of the bone marrow cells into hemophilia A mice partially restored factor VIII (FVIII) production and protected hemophilia A mice from bleeding challenge. Surprisingly, in recipient hemophilia A mice, the donor BM-derived hepatocytes or endothelial cells were rare; the donor-derived mononuclear cells and mesenchymal stromal cells (MSCs) contributed to major factor VIII gene expression and activity.