Transplants for hemophilia

Current research focuses on the task of replacing the function of a deficient gene due to inborn defects such as hemophilia. The ability of hematopoietic cells (HSCs) to permanently reconstitute the hematopoietic and the immune system after transplantation makes these cells a desirable tool for gene therapy approaches. Therefore, HSCs are considered a suitable target cell population in gene therapy for genetic disorders susceptible to bone-marrow transplantation. In the treatment of hemophilia, successful stem-cell transplants have to rely on the ability of stem cells to survive for a long time in the appropriate conditions while expressing the therapeutic product. Recent experiments have shown the importance of the immune response in mediating stem cell graft rejection, demonstrating that cell transplantation for hemophilia is not a simple task. Adult bone marrow cells can be further divided into undifferentiated parenchymal cells, generally referred to as stem cells, and partially differentiated mesenchymal stromal cells (which play a supportive role for the former).