Blood, marrow and cord blood stem cells
Haematopoietic stem cells can be transplanted from bone marrow or collected from peripheral blood after mobilisation or grafted from umbilical cord blood harvests. In children, such transplants can cure a variety of blood, immune and metabolic disorders if a suitable HLA matched donor is available. Strategies to improve transplant outcomes include the production of virus specific T cells and T cells engineered to include a safety gene in case of GVHD.
For a number of conditions, correction of a patient's own stem cells is now a therapeutic option if a transplant donor is not available. We are developing gene addition and gene editing approaches for a number of single gene disorders.