Transplantation and Gene Therapy

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Transplantation and Gene Therapy

Bone marrow transplants may be a cure but currently the risks are too high. Impediments to transplantation include the lack of matched sibling donors and prior transfusions, which have exposed the patient to donor antigens.

The best candidates for bone marrow transplants are children less than 16 years old. Two umbilical cord blood transplantations have been performed that reportedly have not remanifested with sickle cell disease.

Gene therapy may become an option in the future that may alter the expression of the sickle gene.