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This cellular therapy approach involves the transplantation of CD34-selected, T-cell depleted allogeneic hematopoietic stem cells derived from familial haploidentical donors (typically parents). Developed by New York Medical College, the therapy is specifically designed for patients with high-risk sickle cell disease who lack a matched sibling donor. The manufacturing process utilizes the CliniMACS system to enrich for CD34+ progenitor cells while depleting CD3+ T-cells to minimize the risk of graft-versus-host disease (GvHD). This is followed by a controlled T-cell addback to facilitate immune reconstitution and engraftment. The procedure is preceded by a comprehensive conditioning regimen that may include agents such as busulfan, fludarabine, thiotepa, cyclophosphamide, and anti-thymocyte globulin (ATG). The therapeutic objective is to achieve stable donor chimerism, effectively replacing the recipient's sickle-cell-producing bone marrow with healthy donor-derived hematopoiesis.
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