Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

Alexis A. Thompson(Lucile Packard Children's Hospital), Mark C. Walters(Lucile Packard Children's Hospital), Janet L. Kwiatkowski(Lucile Packard Children's Hospital), John E.J. Rasko(Lucile Packard Children's Hospital), Jean‐Antoine Ribeil(Lucile Packard Children's Hospital), Suradej Hongeng(Lucile Packard Children's Hospital), Elisa Magrin(Lucile Packard Children's Hospital), Gary J. Schiller(Lucile Packard Children's Hospital), Emmanuel Payen(Lucile Packard Children's Hospital), Michaëla Semeraro(Lucile Packard Children's Hospital), Despina Moshous(Lucile Packard Children's Hospital), François Lefrère(Lucile Packard Children's Hospital), Hervé Puy(Lucile Packard Children's Hospital), Philippe Bourget(Lucile Packard Children's Hospital), Alessandra Magnani(Lucile Packard Children's Hospital), Laure Caccavelli(Lucile Packard Children's Hospital), Jean‐Sébastien Diana(Lucile Packard Children's Hospital), Felipe Suarez(Lucile Packard Children's Hospital), Fabrice Monpoux(Lucile Packard Children's Hospital), Valentine Brousse(Lucile Packard Children's Hospital), Catherine Poirot(Lucile Packard Children's Hospital), Chantal Brouzes(Lucile Packard Children's Hospital), Jean–François Méritet(Lucile Packard Children's Hospital), Corinne Pondarré(Lucile Packard Children's Hospital), Yves Beuzard(Lucile Packard Children's Hospital), Stany Chrétien(Lucile Packard Children's Hospital), Thibaud Lefèbvre(Lucile Packard Children's Hospital), David T. Teachey(Lucile Packard Children's Hospital), Usanarat Anurathapan(Lucile Packard Children's Hospital), P. Joy Ho(Lucile Packard Children's Hospital), Christof von Kalle(Lucile Packard Children's Hospital), Morris Kletzel(Lucile Packard Children's Hospital), Elliott Vichinsky(Lucile Packard Children's Hospital), Sandeep Soni(Lucile Packard Children's Hospital), Gábor Veres(Lucile Packard Children's Hospital), Olivier Nègre(Lucile Packard Children's Hospital), Robert W. Ross(Lucile Packard Children's Hospital), David Davidson(Lucile Packard Children's Hospital), Alexandria Petrusich(Lucile Packard Children's Hospital), Laura Sandler(Lucile Packard Children's Hospital), Mohammed Asmal(Lucile Packard Children's Hospital), Olivier Hermine(Lucile Packard Children's Hospital), Mariane De Montalembert(Lucile Packard Children's Hospital), Salima Hacein‐Bey‐Abina(Lucile Packard Children's Hospital), Stéphane Blanche(Lucile Packard Children's Hospital), Philippe Leboulch(Lucile Packard Children's Hospital), Marina Cavazzana(Lucile Packard Children's Hospital)
New England Journal of Medicine
April 18, 2018
Cited by 659Open Access
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Abstract

Donor availability and transplantation-related risks limit the broad use of allogeneic


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