Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

A. Thompson, Alexis; C. Walters, Mark; L. Kwiatkowski, Janet; E.J. Rasko, John; Ribeil, Jean‐Antoine; Hongeng, Suradej; Magrin, Elisa; J. Schiller, Gary; Payen, Emmanuel; Semeraro, Michaëla; Moshous, Despina; Lefrère, François; Puy, Hervé; Bourget, Philippe; Magnani, Alessandra; Caccavelli, Laure; Diana, Jean‐Sébastien; Suarez, Felipe; Monpoux, Fabrice; Brousse, Valentine; Poirot, Catherine; Brouzes, Chantal; Méritet, Jean–François; Pondarré, Corinne; Beuzard, Yves; Chrétien, Stany; Lefèbvre, Thibaud; T. Teachey, David; Anurathapan, Usanarat; Joy Ho, P.; von Kalle, Christof; Kletzel, Morris; Vichinsky, Elliott; Soni, Sandeep; Veres, Gábor; Nègre, Olivier; W. Ross, Robert; Davidson, David; Petrusich, Alexandria; Sandler, Laura; Asmal, Mohammed; Hermine, Olivier; De Montalembert, Mariane; Hacein‐Bey‐Abina, Salima; Blanche, Stéphane; Leboulch, Philippe; Cavazzana, Marina

doi:10.1056/nejmoa1705342

Public

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

Published in New England Journal of Medicine • Apr 18, 2018

Authors:

Alexis A. Thompson

Mark C. Walters

Janet L. Kwiatkowski

Abstract

BACKGROUND: ) gene could substitute for long-term red-cell transfusions in a patient with β-thalassemia, we wanted to evaluate the safety and efficacy of such gene therapy in patients with transfusion-dependent β-thalassemia. METHODS: , transfusion requirements, and average vector copy number. RESUL...

Subject

Thalassemia

Transfusion therapy

Medicine

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