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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 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.
Gene therapy: X-SCID transgene leukaemogenicity.
Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, Otsu M, Sorrentino B, Scobie L, Cameron E, Blyth K, Neil J, Abina SH, Cavazzana-Calvo M, Fischer A. Thrasher AJ, et al. Nature. 2006 Sep 21;443(7109):E5-6; discussion E6-7. doi: 10.1038/nature05219. Nature. 2006. PMID: 16988659
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy.
Deichmann A, Hacein-Bey-Abina S, Schmidt M, Garrigue A, Brugman MH, Hu J, Glimm H, Gyapay G, Prum B, Fraser CC, Fischer N, Schwarzwaelder K, Siegler ML, de Ridder D, Pike-Overzet K, Howe SJ, Thrasher AJ, Wagemaker G, Abel U, Staal FJ, Delabesse E, Villeval JL, Aronow B, Hue C, Prinz C, Wissler M, Klanke C, Weissenbach J, Alexander I, Fischer A, von Kalle C, Cavazzana-Calvo M. Deichmann A, et al. J Clin Invest. 2007 Aug;117(8):2225-32. doi: 10.1172/JCI31659. J Clin Invest. 2007. PMID: 17671652 Free PMC article.
Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency.
Neven B, Leroy S, Decaluwe H, Le Deist F, Picard C, Moshous D, Mahlaoui N, Debré M, Casanova JL, Dal Cortivo L, Madec Y, Hacein-Bey-Abina S, de Saint Basile G, de Villartay JP, Blanche S, Cavazzana-Calvo M, Fischer A. Neven B, et al. Blood. 2009 Apr 23;113(17):4114-24. doi: 10.1182/blood-2008-09-177923. Epub 2009 Jan 23. Blood. 2009. PMID: 19168787 Free 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.
Gene therapy of X-linked severe combined immunodeficiency.
Hacein-Bey-Abina S, de Saint Basile G, Cavazzana-Calvo M. Hacein-Bey-Abina S, et al. Methods Mol Biol. 2003;215:247-59. doi: 10.1385/1-59259-345-3:247. Methods Mol Biol. 2003. PMID: 12512304 No abstract available.
199 results