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Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome.
Magnani A, Semeraro M, Adam F, Booth C, Dupré L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Bayford JX, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer A, Six E, Thrasher AJ, Cavazzana M. Magnani A, et al. Nat Med. 2022 Jan;28(1):71-80. doi: 10.1038/s41591-021-01641-x. Epub 2022 Jan 24. Nat Med. 2022. PMID: 35075289 Free PMC article.
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
112 results