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  1. Pai, Sung-Yun; Notarangelo, Luigi Daniele; Harris, Chad; Cattaneo, Federica; Wladkowski, Matthew; Armant, Myriam; Kao, Grace S.; Wielgosz, Matthew; Nienhuis, Arthur W.; Modlich, Ute; Brugman, Martijn; Schambach, Axel; Rothe, Michael; Thornhill, Susannah I; Gaspar, H. Bobby; Parsley, Kathryn; Cooray, Samantha; Grassman, Elke; Bushman, Frederic; Shupien, Sally; Oleastro, Matias; Hacein-Bey-Abina, Salima; Fischer, Alain; Cavazzana-Calvo, Marina; [...]

    Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

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    American Society of Hematology, 2011

    Published in: Blood