Article
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.
The New England journal of medicine - 19 Apr 2018
Thompson Alexis A, Walters Mark C, Kwiatkowski Janet, Rasko John E J, Ribeil Jean-Antoine, Hongeng Suradej, Magrin Elisa, Schiller Gary J, Payen Emmanuel, Semeraro Michaela, Moshous Despina, Lefrere Francois, Puy Hervé, Bourget Philippe, Magnani Alessandra, Caccavelli Laure, Diana Jean-Sébastien, Suarez Felipe, Monpoux Fabrice, Brousse Valentine, Poirot Catherine, Brouzes Chantal, Meritet Jean-François, Pondarré Corinne, Beuzard Yves, Chrétien Stany, Lefebvre Thibaud, Teachey David T, Anurathapan Usanarat, Ho P Joy, von Kalle Christof, Kletzel Morris, Vichinsky Elliott, Soni Sandeep, Veres Gabor, Negre Olivier, Ross Robert W, Davidson David, Petrusich Alexandria, Sandler Laura, Asmal Mohammed, Hermine Olivier, De Montalembert Mariane, Hacein-Bey-Abina Salima, Blanche Stéphane, Leboulch Philippe, Cavazzana Marina
Abstract excerpt
BACKGROUND: Donor availability and transplantation-related risks limit the broad use of allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β-thalassemia. After previously establishing that lentiviral transfer of a marked β-globin (βA-T87Q) gene could substitute for long-term red-cell transfusions in a patient with β-thalassemia, we wanted to evaluate the safety and efficacy of...
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