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2. Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia. Thompson AA; Walters MC; Kwiatkowski J; Rasko JEJ; Ribeil JA; Hongeng S; Magrin E; Schiller GJ; Payen E; Semeraro M; Moshous D; Lefrere F; Puy H; Bourget P; Magnani A; Caccavelli L; Diana JS; Suarez F; Monpoux F; Brousse V; Poirot C; Brouzes C; Meritet JF; Pondarré C; Beuzard Y; Chrétien S; Lefebvre T; Teachey DT; Anurathapan U; Ho PJ; von Kalle C; Kletzel M; Vichinsky E; Soni S; Veres G; Negre O; Ross RW; Davidson D; Petrusich A; Sandler L; Asmal M; Hermine O; De Montalembert M; Hacein-Bey-Abina S; Blanche S; Leboulch P; Cavazzana M N Engl J Med; 2018 Apr; 378(16):1479-1493. PubMed ID: 29669226 [TBL] [Abstract][Full Text] [Related]
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