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CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells.
Brault J, Liu T, Liu S, Lawson A, Choi U, Kozhushko N, Bzhilyanskaya V, Pavel-Dinu M, Meis RJ, Eckhaus MA, Burkett SS, Bosticardo M, Kleinstiver BP, Notarangelo LD, Lazzarotto CR, Tsai SQ, Wu X, Dahl GA, Porteus MH, Malech HL, De Ravin SS. Brault J, et al. Front Immunol. 2023 Jan 4;13:1067417. doi: 10.3389/fimmu.2022.1067417. eCollection 2022. Front Immunol. 2023. PMID: 36685559 Free PMC article.
CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes.
Brault J, Liu T, Bello E, Liu S, Sweeney CL, Meis RJ, Koontz S, Corsino C, Choi U, Vayssiere G, Bosticardo M, Dowdell K, Lazzarotto CR, Clark AB, Notarangelo LD, Ravell JC, Lenardo MJ, Kleinstiver BP, Tsai SQ, Wu X, Dahl GA, Malech HL, De Ravin SS. Brault J, et al. Blood. 2021 Dec 30;138(26):2768-2780. doi: 10.1182/blood.2021011192. Blood. 2021. PMID: 34086870 Free PMC article.
MAGT1 messenger RNA-corrected autologous T and natural killer cells for potential cell therapy in X-linked immunodeficiency with magnesium defect, Epstein-Barr virus infection and neoplasia disease.
Brault J, Meis RJ, Li L, Bello E, Liu T, Sweeney CL, Koontz SM, Dowdell K, Theobald N, Lee J, Allen C, Clark AB, Ravell JC, Lenardo MJ, Dahl GA, Malech HL, De Ravin SS. Brault J, et al. Cytotherapy. 2021 Mar;23(3):203-210. doi: 10.1016/j.jcyt.2020.08.013. Epub 2020 Oct 10. Cytotherapy. 2021. PMID: 33051095
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency.
De Ravin SS, Liu S, Sweeney CL, Brault J, Whiting-Theobald N, Ma M, Liu T, Choi U, Lee J, O'Brien SA, Quackenbush P, Estwick T, Karra A, Docking E, Kwatemaa N, Guo S, Su L, Sun Z, Zhou S, Puck J, Cowan MJ, Notarangelo LD, Kang E, Malech HL, Wu X. De Ravin SS, et al. Nat Commun. 2022 Jun 28;13(1):3710. doi: 10.1038/s41467-022-31344-x. Nat Commun. 2022. PMID: 35764638 Free PMC article. Clinical Trial.
Impact of CXCR4 inhibition on FLT3-ITD-positive human AML blasts.
Jacobi A, Thieme S, Lehmann R, Ugarte F, Malech HL, Koch S, Thiede C, Müller K, Bornhäuser M, Ryser M, Brenner S. Jacobi A, et al. Exp Hematol. 2010 Mar;38(3):180-90. doi: 10.1016/j.exphem.2009.12.003. Epub 2009 Dec 24. Exp Hematol. 2010. PMID: 20035824 Free PMC article.
Successful SCID gene therapy in infant with disseminated BCG.
Maron G, Kaste S, Bahrami A, Neel M, Malech HL, Puck JM, Cowan MJ, Gottschalk S, Mamcarz E. Maron G, et al. J Allergy Clin Immunol Pract. 2021 Feb;9(2):993-995.e1. doi: 10.1016/j.jaip.2020.09.004. Epub 2020 Sep 16. J Allergy Clin Immunol Pract. 2021. PMID: 32949809 Free PMC article. No abstract available.
NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease.
De Ravin SS, Brault J, Meis RJ, Li L, Theobald N, Bonifacino AC, Lei H, Liu TQ, Koontz S, Corsino C, Zarakas MA, Desai JV, Clark AB, Choi U, Metzger ME, West K, Highfill SL, Kang E, Kuhns DB, Lionakis MS, Stroncek DF, Dunbar CE, Tisdale JF, Donahue RE, Dahl GA, Malech HL. De Ravin SS, et al. Blood Adv. 2020 Dec 8;4(23):5976-5987. doi: 10.1182/bloodadvances.2020003224. Blood Adv. 2020. PMID: 33284949 Free PMC article.