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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. Among authors: alexander i. Science. 2003 Oct 17;302(5644):415-9. doi: 10.1126/science.1088547. Science. 2003. PMID: 14564000
Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia.
Ginn SL, Curtin JA, Kramer B, Smyth CM, Wong M, Kakakios A, McCowage GB, Watson D, Alexander SI, Latham M, Cunningham SC, Zheng M, Hobson L, Rowe PB, Fischer A, Cavazzana-Calvo M, Hacein-Bey-Abina S, Alexander IE. Ginn SL, et al. Among authors: alexander si, alexander ie. Med J Aust. 2005 May 2;182(9):458-63. doi: 10.5694/j.1326-5377.2005.tb06785.x. Med J Aust. 2005. PMID: 15865589
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. Among authors: alexander i. J Clin Invest. 2007 Aug;117(8):2225-32. doi: 10.1172/JCI31659. J Clin Invest. 2007. PMID: 17671652 Free PMC article.
Lymphoid regeneration from gene-corrected SCID-X1 subject-derived iPSCs.
Menon T, Firth AL, Scripture-Adams DD, Galic Z, Qualls SJ, Gilmore WB, Ke E, Singer O, Anderson LS, Bornzin AR, Alexander IE, Zack JA, Verma IM. Menon T, et al. Cell Stem Cell. 2015 Apr 2;16(4):367-72. doi: 10.1016/j.stem.2015.02.005. Epub 2015 Mar 12. Cell Stem Cell. 2015. PMID: 25772073 Free PMC article.
Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency.
Ginn SL, Hallwirth CV, Liao SHY, Teber ET, Arthur JW, Wu J, Lee HC, Tay SS, Hu M, Reddel RR, McCormack MP, Thrasher AJ, Cavazzana M, Alexander SI, Alexander IE. Ginn SL, et al. Among authors: alexander si, alexander ie. Mol Ther Nucleic Acids. 2017 Mar 17;6:1-14. doi: 10.1016/j.omtn.2016.11.011. Epub 2016 Dec 10. Mol Ther Nucleic Acids. 2017. PMID: 28325276 Free PMC article.
384 results