A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Hacein‐Bey‐Abina, Salima; Pai, Sung‐Yun; Bobby Gaspar, H.; Armant, Myriam; C. Berry, Charles; Blanche, Stéphane; Bleesing, Jack; Blondeau, Johanna; de Boer, Helen; Buckland, Karen; Caccavelli, Laure; Cros, Guilhem; De Oliveira, Satiro; S. Fernández, Karen; Guo, Dongjing; E. Harris, Chad; Hopkins, Gregory; Lehmann, Leslie; Lim, Annick; B. London, Wendy; C.M. van der Loo, Johannes; Malani, Nirav; Male, Frances; Malik, Punam; Angélica Marinovic, María; McNicol, Anne-Marie; Moshous, Despina; Neven, Bénédicte; Oleastro, Matías; Pïcard, Capucine; Ritz, Jerome; Rivat, Christine; Schambach, Axel; L. Shaw, Kit; A. Sherman, Eric; E. Silberstein, Leslie; Six, Emmanuelle; Touzot, Fabien; V. Tsytsykova, Alla; Xu‐Bayford, Jinhua; Baum, Christopher; D. Bushman, Frederic; Fischer, Alain; B. Kohn, Donald; H. Filipovich, Alexandra; D. Notarangelo, Luigi; Cavazzana, Marina; A. Williams, David; J. Thrasher, Adrian

doi:10.1056/nejmoa1404588

Public

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Published in New England Journal of Medicine • Oct 8, 2014

Authors:

Salima Hacein‐Bey‐Abina

Sung‐Yun Pai

H. Bobby Gaspar

Abstract

BACKGROUND: In previous clinical trials involving children with X-linked severe combined immunodeficiency (SCID-X1), a Moloney murine leukemia virus-based γ-retrovirus vector expressing interleukin-2 receptor γ-chain (γc) complementary DNA successfully restored immunity in most patients but resulted...

Subject

Retrovirus

Medicine

Severe combined immunodeficiency

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