Waseem Qasim Research Group

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 where matched donors are not available are being developed

 

For a number of conditions, correction of a patient's own stem cells is being investigated as a therapeutic option if a transplant donor is not available. Early approaches have used viral vectors to 'add' and extra copies of defective genes but genome editing is now making it feasible to correct the underlying DNA codes back to normal. 

 

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