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Allogeneic HLA-identical bone marrow-derived mesenchymal stem cells (MSCs) is an investigational cell therapy being evaluated for the treatment of osteogenesis imperfecta (OI), specifically type III OI caused by mutations in the COL1A1 or COL1A2 genes. The therapy involves the repeated intravenous infusion of non-mutated, histocompatible (HLA-identical or sharing at least 5 out of 6 HLA antigens) MSCs obtained from healthy donor bone marrow. These cells are intended to home to the bone, engraft, and differentiate into functional osteoblasts that produce normal type I collagen, thereby improving bone strength and reducing fracture frequency in pediatric patients. A key feature of this protocol is the absence of immunosuppressive therapy, leveraging the low immunogenicity and immunomodulatory properties of MSCs. The therapy is currently being investigated in a Phase 1 clinical trial in Spain led by Itziar Astigarraga Aguirre.
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