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Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.
Hacein-Bey Abina S, Gaspar HB, Blondeau J, Caccavelli L, Charrier S, Buckland K, Picard C, Six E, Himoudi N, Gilmour K, McNicol AM, Hara H, Xu-Bayford J, Rivat C, Touzot F, Mavilio F, Lim A, Treluyer JM, Héritier S, Lefrère F, Magalon J, Pengue-Koyi I, Honnet G, Blanche S, Sherman EA, Male F, Berry C, Malani N, Bushman FD, Fischer A, Thrasher AJ, Galy A, Cavazzana M. Hacein-Bey Abina S, et al. JAMA. 2015 Apr 21;313(15):1550-63. doi: 10.1001/jama.2015.3253. JAMA. 2015. PMID: 25898053 Free PMC article. Clinical Trial.
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.
Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP, Thrasher AJ, Wulffraat N, Sorensen R, Dupuis-Girod S, Fischer A, Davies EG, Kuis W, Leiva L, Cavazzana-Calvo M. Hacein-Bey-Abina S, et al. N Engl J Med. 2002 Apr 18;346(16):1185-93. doi: 10.1056/NEJMoa012616. N Engl J Med. 2002. PMID: 11961146 Free article. Clinical Trial.
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.
Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A, Cavazzana-Calvo M. Hacein-Bey-Abina S, et al. Science. 2003 Oct 17;302(5644):415-9. doi: 10.1126/science.1088547. Science. 2003. PMID: 14564000
Gene therapy of X-linked severe combined immunodeficiency.
Cavazzana-Calvo M, Hacein-Bey-Abina S, Fischer A. Cavazzana-Calvo M, et al. Curr Opin Allergy Clin Immunol. 2002 Dec;2(6):507-9. doi: 10.1097/00130832-200212000-00005. Curr Opin Allergy Clin Immunol. 2002. PMID: 14752333 Review.
Impact of HLA matching on outcome of hematopoietic stem cell transplantation in children with inherited diseases: a single-center comparative analysis of genoidentical, haploidentical or unrelated donors.
Caillat-Zucman S, Le Deist F, Haddad E, Gannagé M, Dal Cortivo L, Jabado N, Hacein-Bey-Abina S, Blanche S, Casanova JL, Fischer A, Cavazzana-Calvo M. Caillat-Zucman S, et al. Bone Marrow Transplant. 2004 Jun;33(11):1089-95. doi: 10.1038/sj.bmt.1704510. Bone Marrow Transplant. 2004. PMID: 15077132
Gene therapy for immunodeficiency diseases.
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Fischer A, et al. Semin Hematol. 2004 Oct;41(4):272-8. doi: 10.1053/j.seminhematol.2004.07.003. Semin Hematol. 2004. PMID: 15508112 Review.
112 results