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This investigational cell therapy, developed by St. Jude Children's Research Hospital, consists of ex vivo expanded, donor-derived, gene-marked bone marrow stromal cells (also known as mesenchymal stromal cells or MSCs). The therapy is designed for children with osteodysplasia, specifically those with osteogenesis imperfecta, who have previously undergone an allogeneic bone marrow transplant. The active ingredient comprises multipotent progenitor cells that are expanded in culture and then infused into the patient. These cells are capable of engrafting in the skeletal system and differentiating into functional bone-forming cells (osteoblasts), thereby potentially ameliorating skeletal dysplasias by providing healthy osteoprogenitor cells to produce normal bone matrix.
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