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Boulad, Farid; Mansilla-Soto, Jorge; Cabriolu, Annalisa; Rivière, Isabelle; Sadelain, Michel
Hematology/oncology clinics of North America, 04/2018, Volume: 32, Issue: 2Journal Article
The β-thalassemias are inherited blood disorders that result from insufficient production of the β-chain of hemoglobin. More than 200 different mutations have been identified. β-Thalassemia major requires life-long transfusions. The only cure for severe β-thalassemia is to provide patients with hematopoietic stem cells. Globin gene therapy promises a curative autologous stem cell transplantation without the immunologic complications of allogeneic transplantation. The future directions of gene therapy include enhancement of lentiviral vector-based approaches, fine tuning of the conditioning regimen, and the design of safer vectors. Progress in genetic engineering bodes well for finding a cure for severe globin disorders.
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