Gene Therapy in a Patient with Sickle Cell Disease
Gene Therapy in a Patient with Sickle Cell Disease
About this item
Full title
Author / Creator
Ribeil, Jean-Antoine , Hacein-Bey-Abina, Salima , Payen, Emmanuel , Magnani, Alessandra , Semeraro, Michaela , Magrin, Elisa , Caccavelli, Laure , Neven, Benedicte , Bourget, Philippe , El Nemer, Wassim , Bartolucci, Pablo , Weber, Leslie , Puy, Hervé , Meritet, Jean-François , Grevent, David , Beuzard, Yves , Chrétien, Stany , Lefebvre, Thibaud , Ross, Robert W , Negre, Olivier , Veres, Gabor , Sandler, Laura , Soni, Sandeep , de Montalembert, Mariane , Blanche, Stéphane , Leboulch, Philippe and Cavazzana, Marina
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
A boy with hydroxyurea-refractory sickle cell anemia underwent bone marrow transplantation with autologous hematopoietic stem cells transduced by a lentivirus to express an antisickling β-globin variant. No sickle cell crises occurred in the following 15 months.
Sickle cell disease is among the most prevalent inherited monogenic disorders. Appro...
Alternative Titles
Full title
Gene Therapy in a Patient with Sickle Cell Disease
Authors, Artists and Contributors
Author / Creator
Hacein-Bey-Abina, Salima
Payen, Emmanuel
Magnani, Alessandra
Semeraro, Michaela
Magrin, Elisa
Caccavelli, Laure
Neven, Benedicte
Bourget, Philippe
El Nemer, Wassim
Bartolucci, Pablo
Weber, Leslie
Puy, Hervé
Meritet, Jean-François
Grevent, David
Beuzard, Yves
Chrétien, Stany
Lefebvre, Thibaud
Ross, Robert W
Negre, Olivier
Veres, Gabor
Sandler, Laura
Soni, Sandeep
de Montalembert, Mariane
Blanche, Stéphane
Leboulch, Philippe
Cavazzana, Marina
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_hal_primary_oai_HAL_inserm_01834781v1
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_hal_primary_oai_HAL_inserm_01834781v1
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1609677
