Frequently asked question: Can a stem cell and bone marrow transplantation cure sickle cell disease?

Sickle cell disease is a hereditary condition that can only be cured by replacing a patient’s bone marrow with donor bone marrow.
This is called a stem cell or bone marrow transplant, which is an intensive and complex procedure that replaces the patient’s entire red blood cell production infrastructure with that of a donor. The new produced red blood cells will not have hemoglobin with an abnormal beta globin chain, but instead have normal beta globin chains, which means that normal hemoglobin A1 will be produced. In the Netherlands, there is considerable experience with this kind of transplant. However, it is rarely used in cases of sickle cell disease, because the adverse effects and risks still outweigh the benefits.

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