Hematopoietic stem cell transplantation for the treatment of genetic blood cell-related diseases

The aim of this study is to assess whether transplantation of allogeneic hematopoietic stem cells (HSCs) can be used as a treatment for several genetic diseases affecting blood cells. This study will include patients without an HLA-matched sibling donor. For such patients, HSCs will be obtained from matched unrelated donors or unrelated umbilical cord blood.