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 …