Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

Author:

Thompson Alexis A.1,Walters Mark C.1,Kwiatkowski Janet1,Rasko John E.J.1,Ribeil Jean-Antoine1,Hongeng Suradej1,Magrin Elisa1,Schiller Gary J.1,Payen Emmanuel1,Semeraro Michaela1,Moshous Despina1,Lefrere Francois1,Puy Hervé1,Bourget Philippe1,Magnani Alessandra1,Caccavelli Laure1,Diana Jean-Sébastien1,Suarez Felipe1,Monpoux Fabrice1,Brousse Valentine1,Poirot Catherine1,Brouzes Chantal1,Meritet Jean-François1,Pondarré Corinne1,Beuzard Yves1,Chrétien Stany1,Lefebvre Thibaud1,Teachey David T.1,Anurathapan Usanarat1,Ho P. Joy1,von Kalle Christof1,Kletzel Morris1,Vichinsky Elliott1,Soni Sandeep1,Veres Gabor1,Negre Olivier1,Ross Robert W.1,Davidson David1,Petrusich Alexandria1,Sandler Laura1,Asmal Mohammed1,Hermine Olivier1,De Montalembert Mariane1,Hacein-Bey-Abina Salima1,Blanche Stéphane1,Leboulch Philippe1,Cavazzana Marina1

Affiliation:

1. From the Ann and Robert H. Lurie Children’s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children’s Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children’s Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) — all in California; Children’s Hospital of Philadelphia and University of Pennsylvania Perelman School...

Funder

bluebird bio

National Center for Advancing Translational Sciences

Assistance Publique - Hôpitaux de Paris

Institut National de la Santé et de la Recherche Médicale

Commissariat à l'Énergie Atomique et aux Énergies Alternatives

Agence Nationale de la Recherche

Publisher

Massachusetts Medical Society

Subject

General Medicine

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