Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

Hacein-Bey Abina, Salima ; Gaspar, H. Bobby ; Blondeau, Johanna ; Caccavelli, Laure ; Charrier, Sabine ; Buckland, Karen ; Picard, Capucine ; Six, Emmanuelle ; Himoudi, Nourredine ; Gilmour, Kimberly ; McNicol, Anne-Marie ; Hara, Havinder ; Xu-Bayford, Jinhua ; Rivat, Christine ; Touzot, Fabien ; Mavilio, Fulvio ; Lim, Annick ; Treluyer, Jean-Marc ; Héritier, Sébastien ; Lefrère, Francois ; Magalon, Jeremy ; Pengue-Koyi, Isabelle ; Honnet, Géraldine ; Blanche, Stéphane ; Sherman, Eric A ; Male, Frances ; Berry, Charles ; Malani, Nirav ; Bushman, Frederic D ; Fischer, Alain ; Thrasher, Adrian J ; Galy, Anne ; Cavazzana, Marina

JAMA : the journal of the American Medical Association, 2015-04, Vol.313 (15), p.1550-1563 [Periódico revisado por pares]

United States: American Medical Association

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