Article
Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1.
Blood - 4 Jun 2015
Touzot Fabien, Moshous Despina, Creidy Rita, Neven Bénédicte, Frange Pierre, Cros Guilhem, Caccavelli Laure, Blondeau Johanna, Magnani Alessandra, Luby Jean-Marc, Ternaux Brigitte, Picard Capucine, Blanche Stéphane, Fischer Alain, Hacein-Bey-Abina Salima, Cavazzana Marina
Abstract excerpt
During the last decade, gene therapy via ex vivo gene transfer into autologous hematopoietic stem cells has emerged as a convincing therapy for severe combined immunodeficiency caused by ILR2G mutation (SCID-X1) despite the occurrence of genotoxicity caused by the integration of first-generation...
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