Article
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.
The New England journal of medicine - 9 Oct 2014
Hacein-Bey-Abina Salima, Pai Sung-Yun, Gaspar H Bobby, Armant Myriam, Berry Charles C, Blanche Stephane, Bleesing Jack, Blondeau Johanna, de Boer Helen, Buckland Karen F, Caccavelli Laure, Cros Guilhem, De Oliveira Satiro, Fernández Karen S, Guo Dongjing, Harris Chad E, Hopkins Gregory, Lehmann Leslie E, Lim Annick, London Wendy B, van der Loo Johannes C M, Malani Nirav, Male Frances, Malik Punam, Marinovic M Angélica, McNicol Anne-Marie, Moshous Despina, Neven Benedicte, Oleastro Matías, Picard Capucine, Ritz Jerome, Rivat Christine, Schambach Axel, Shaw Kit L, Sherman Eric A, Silberstein Leslie E, Six Emmanuelle, Touzot Fabien, Tsytsykova Alla, Xu-Bayford Jinhua, Baum Christopher, Bushman Frederic D, Fischer Alain, Kohn Donald B, Filipovich Alexandra H, Notarangelo Luigi D, Cavazzana Marina, Williams David A, Thrasher Adrian J
Abstract excerpt
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 in vector-induced leukemia through enhancer-mediated mutagenesis in 25% of patients. We assessed the efficacy and...
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