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Allogeneic hematopoietic stem cell transplantation (HSCT) is a cellular therapy utilized as a curative treatment for malignant infantile osteopetrosis (MIOP), a rare and fatal genetic disorder characterized by dysfunctional osteoclasts and impaired bone remodeling. In the protocol developed by St. Jude Children's Research Hospital, hematopoietic stem cells are typically sourced from a haploidentical (partially matched) parental donor when a matched sibling is unavailable. These donor cells are processed using the Miltenyi Biotec CliniMACS device for T-cell depletion to facilitate engraftment while minimizing the risk of graft-versus-host disease (GVHD). Following a conditioning regimen, the infused stem cells engraft in the recipient's bone marrow and differentiate into functional osteoclasts, which restore the bone's ability to regulate resorption and remodeling.
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