At the moment, the only way to cure sickle cell anemia and Diamond-Blackfan anemia syndrome is by doing a stem cell transplant with blood stem cells from a donor. This is a procedure that is not without risk, because the donated blood stem cells can attack the body of the recepient if there is not a full match.
With help of the findings in research & repair, we are trying to grow genetically corrected induced pluripotent stem cells (iPSCs) of a patient with sickle cell anemia or Diamond-Blackfan anemia syndrome into a supply of healthy blood stem cells. This way a patient can receive blood stem cells of their own (repaired) DNA that will match with their immune system for 100%. This reduces the risk of rejection between the body and the tranplanted blood stem cells and increases the probability to cure the patient.
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