Gene Therapy in Patients with Transfusion-Dependent [beta]-Thalassemia
Gene Therapy in Patients with Transfusion-Dependent [beta]-Thalassemia
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Author / Creator
Thompson, Alexis A , Walters, Mark C , Kwiatkowski, Janet , Rasko, John EJ , Ribeil, Jean-Antoine , Hongeng, Suradej , Magrin, Elisa , Schiller, Gary J , Payen, Emmanuel , Semeraro, Michaela , Moshous, Despina , Lefrere, Francois , Puy, Hervé , Bourget, Philippe , Magnani, Alessandra , Caccavelli, Laure , Diana, Jean-Sébastien , Suarez, Felipe , Monpoux, Fabrice , Brousse, Valentine , Poirot, Catherine , Brouzes, Chantal , Meritet, Jean-François , Pondarré, Corinne , Beuzard, Yves , Chrétien, Stany , Lefebvre, Thibaud , Teachey, David T , Anurathapan, Usanarat , Ho, P Joy , von Kalle, Christof , Kletzel, Morris , Vichinsky, Elliott , Soni, Sandeep , Veres, Gabor , Negre, Olivier , Ross, Robert W , Davidson, David , Petrusich, Alexandria , Sandler, Laura , Asmal, Mohammed , Hermine, Olivier , De Montalembert, Mariane , Hacein-Bey-Abina, Salima , Blanche, Stéphane , Leboulch, Philippe and Cavazzana, Marina
Publisher
Boston: Massachusetts Medical Society
Journal title
Language
English
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Publication information
Publisher
Boston: Massachusetts Medical Society
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Scope and Contents
Contents
Background Donor availability and transplantation-related risks limit the broad use of allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β-thalassemia. After previously establishing that lentiviral transfer of a marked β-globin (βA-T87Q) gene could substitute for long-term red-cell transfusions in a patient with β...
Alternative Titles
Full title
Gene Therapy in Patients with Transfusion-Dependent [beta]-Thalassemia
Authors, Artists and Contributors
Author / Creator
Walters, Mark C
Kwiatkowski, Janet
Rasko, John EJ
Ribeil, Jean-Antoine
Hongeng, Suradej
Magrin, Elisa
Schiller, Gary J
Payen, Emmanuel
Semeraro, Michaela
Moshous, Despina
Lefrere, Francois
Puy, Hervé
Bourget, Philippe
Magnani, Alessandra
Caccavelli, Laure
Diana, Jean-Sébastien
Suarez, Felipe
Monpoux, Fabrice
Brousse, Valentine
Poirot, Catherine
Brouzes, Chantal
Meritet, Jean-François
Pondarré, Corinne
Beuzard, Yves
Chrétien, Stany
Lefebvre, Thibaud
Teachey, David T
Anurathapan, Usanarat
Ho, P Joy
von Kalle, Christof
Kletzel, Morris
Vichinsky, Elliott
Soni, Sandeep
Veres, Gabor
Negre, Olivier
Ross, Robert W
Davidson, David
Petrusich, Alexandria
Sandler, Laura
Asmal, Mohammed
Hermine, Olivier
De Montalembert, Mariane
Hacein-Bey-Abina, Salima
Blanche, Stéphane
Leboulch, Philippe
Cavazzana, Marina
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Primary Identifiers
Record Identifier
TN_cdi_proquest_journals_2036803057
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_2036803057
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1705342
