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This allogeneic hematopoietic stem cell transplantation (HSCT) protocol is being evaluated by Columbia University for the treatment of sickle cell disease and beta thalassemia. The therapeutic approach utilizes a moderately ablative conditioning regimen—comprising busulfan, fludarabine, and alemtuzumab—to prepare the patient's bone marrow for the infusion of healthy hematopoietic stem cells obtained from matched sibling or unrelated donors. By establishing donor chimerism, the procedure aims to replace the patient's defective erythroid lineage with healthy donor cells that produce functional hemoglobin, potentially curing the underlying genetic disorder. The regimen is designed to achieve successful engraftment with reduced toxicity compared to traditional high-dose myeloablative conditioning.
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