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Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

2018649 citationsJournal Articlebronze Open Access

Authors

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

Abstract

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

Topics & Keywords

Hemoglobinopathies and Related Disorders Erythrocyte Function and Pathophysiology Parvovirus B19 Infection Studies

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: New England Journal of Medicine

Volume 378, Issue 16, pp. 1479-1493

DOI: 10.1056/nejmoa1705342

Field-Weighted Citation Impact: 53.14

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia | Researchclopedia

· 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