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Page 1
Evolving Gene Therapy in Primary Immunodeficiency.
Thrasher AJ, Williams DA. Thrasher AJ, et al. Mol Ther. 2017 May 3;25(5):1132-1141. doi: 10.1016/j.ymthe.2017.03.018. Epub 2017 Mar 31. Mol Ther. 2017. PMID: 28366768 Free PMC article. Review.
Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells.
Qasim W, Zhan H, Samarasinghe S, Adams S, Amrolia P, Stafford S, Butler K, Rivat C, Wright G, Somana K, Ghorashian S, Pinner D, Ahsan G, Gilmour K, Lucchini G, Inglott S, Mifsud W, Chiesa R, Peggs KS, Chan L, Farzeneh F, Thrasher AJ, Vora A, Pule M, Veys P. Qasim W, et al. Sci Transl Med. 2017 Jan 25;9(374):eaaj2013. doi: 10.1126/scitranslmed.aaj2013. Sci Transl Med. 2017. PMID: 28123068
Autoinflammatory periodic fever, immunodeficiency, and thrombocytopenia (PFIT) caused by mutation in actin-regulatory gene WDR1.
Standing AS, Malinova D, Hong Y, Record J, Moulding D, Blundell MP, Nowak K, Jones H, Omoyinmi E, Gilmour KC, Medlar A, Stanescu H, Kleta R, Anderson G, Nanthapisal S, Gomes SM, Klein N, Eleftheriou D, Thrasher AJ, Brogan PA. Standing AS, et al. J Exp Med. 2017 Jan;214(1):59-71. doi: 10.1084/jem.20161228. Epub 2016 Dec 19. J Exp Med. 2017. PMID: 27994071 Free PMC article.
Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions.
Weisser M, Demel UM, Stein S, Chen-Wichmann L, Touzot F, Santilli G, Sujer S, Brendel C, Siler U, Cavazzana M, Thrasher AJ, Reichenbach J, Essers MAG, Schwäble J, Grez M. Weisser M, et al. J Allergy Clin Immunol. 2016 Jul;138(1):219-228.e9. doi: 10.1016/j.jaci.2015.11.028. Epub 2016 Feb 4. J Allergy Clin Immunol. 2016. PMID: 26853280