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Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial.
Schmidt M, Hacein-Bey-Abina S, Wissler M, Carlier F, Lim A, Prinz C, Glimm H, Andre-Schmutz I, Hue C, Garrigue A, Le Deist F, Lagresle C, Fischer A, Cavazzana-Calvo M, von Kalle C. Schmidt M, et al. Blood. 2005 Apr 1;105(7):2699-706. doi: 10.1182/blood-2004-07-2648. Epub 2004 Dec 7. Blood. 2005. PMID: 15585650 Free article. Clinical Trial.
Failure of SCID-X1 gene therapy in older patients.
Thrasher AJ, Hacein-Bey-Abina S, Gaspar HB, Blanche S, Davies EG, Parsley K, Gilmour K, King D, Howe S, Sinclair J, Hue C, Carlier F, von Kalle C, de Saint Basile G, le Deist F, Fischer A, Cavazzana-Calvo M. Thrasher AJ, et al. Blood. 2005 Jun 1;105(11):4255-7. doi: 10.1182/blood-2004-12-4837. Epub 2005 Feb 1. Blood. 2005. PMID: 15687233 Free article.
Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs.
Six E, Guilloux A, Denis A, Lecoules A, Magnani A, Vilette R, Male F, Cagnard N, Delville M, Magrin E, Caccavelli L, Roudaut C, Plantier C, Sobrino S, Gregg J, Nobles CL, Everett JK, Hacein-Bey-Abina S, Galy A, Fischer A, Thrasher AJ, André I, Cavazzana M, Bushman FD. Six E, et al. Blood. 2020 Apr 9;135(15):1219-1231. doi: 10.1182/blood.2019002350. Blood. 2020. PMID: 32040546 Free PMC article. Clinical Trial.
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.
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.
Gene therapy of metabolic diseases.
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Fischer A, et al. J Inherit Metab Dis. 2006 Apr-Jun;29(2-3):409-12. doi: 10.1007/s10545-006-0270-7. J Inherit Metab Dis. 2006. PMID: 16763910 Review.
112 results