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Rivista di Biologia Molecolare e Biotecnologie
Indexed/Abstracted in: EMBASE, Science Citation Index Expanded (SciSearch), Scopus
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ORIGINAL ARTICLES TRENDS IN MOLECULAR DIAGNOSIS AND THERAPY OF β-THALASSEMIA AND SICKLE CELL ANEMIA
Minerva Biotecnologica 2003 June;15(2):99-105
Bone marrow transplantation for β-thalassemia
Giardini C., Lucarelli G.
Hematology/Oncology Department and Bone Marrow Transplant Center, “San Salvatore” General Hospital, Pesaro, Italy
β-thalassemia is a common genetic disease worldwide. Supportive therapies such as chronic lifelong transfusions and iron chelation have significantly ameliorated clinical manifestations of this disease, but cannot eliminate disease and treatment related complications that result in end-organ damage. Allogeneic bone marrow transplantation (BMT) is the only cure for patients with hemoglobinopathies. Results of transplants have steadily improved over the last decades due to effective control of transplant related complications and development of new preparative regimens. Our understandings of mixed chimerism (MC) in patients with hemoglobinopathies provide a rational for the use of less intensive conditioning regimens and gene therapy in these disorders. This review summarizes the current status of BMT for thalassemia major.