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Hematol. Oncol. Clin. North Am. · Dec 2010
ReviewAllogeneic cellular gene therapy for hemoglobinopathies.
- Javid Gaziev and Guido Lucarelli.
- International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinico Tor Vergata, Viale Oxford 81, Rome, Italy. j.gaziev@fondazioneime.org
- Hematol. Oncol. Clin. North Am. 2010 Dec 1; 24 (6): 1145-63.
AbstractHematopoietic stem cell transplantation (HSCT) offers potentially curative therapy for patients with thalassemia major and sickle cell disease (SCD). Current myeloablative treatment protocols allow the cure of 78% to 90% of patients with thalassemia and 72% to 96% with SCD, depending on disease status at the time of transplantation. The major limitation to successful transplantation is the lack of a suitable HLA-matched family donor. Unrelated donor HSCT is now extensively used to treat thalassemia, with results similar to those obtained following transplantation using HLA-matched sibling donors. Patients who lack a matched related or unrelated donor can now benefit from successful transplantation using haploidentical donors.Copyright © 2010 Elsevier Inc. All rights reserved.
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