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Gene Replacement Therapy

Dr. Hans-Peter Kiem, a medical oncologist and scientific investigator at the Fred Hutchinson Cancer Research Center, is researching another alternative known as gene replacement therapy for patients diagnosed with FA in complementation groups A and C. This type of therapy uses the patients own blood cells that have been corrected for either the A or C complementation group defect.

More specifically, the FA patient’s bone marrow cells are collected and a correct copy of the FA gene that was mutated is stably transferred to the cells.  These “corrected” bone marrow cells are then transplanted back into the patient. Because these cells come from the patient, the risk of an immune reaction, like GVHD or graft rejection, is lower.  Also, the corrected cells have an advantage to survive over non-corrected cells once the transplant is complete. The hope is that this survival advantage will allow corrected cells to continue supporting the bone marrow and blood system of the patient and decrease the risk of aplastic anemia relapse.