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How I manage patients with Wiskott Aldrich syndrome.
Rivers E, Worth A, Thrasher AJ, Burns SO. Rivers E, et al. Br J Haematol. 2019 May;185(4):647-655. doi: 10.1111/bjh.15831. Epub 2019 Mar 12. Br J Haematol. 2019. PMID: 30864154 Free PMC article. Review.
Progress in lentiviral vector technologies.
Thrasher AJ. Thrasher AJ. Hum Gene Ther. 2013 Feb;24(2):117-8. doi: 10.1089/hum.2013.2500. Hum Gene Ther. 2013. PMID: 23421453 No abstract available.
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.
Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Tremblay JP, et al. Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. Mol Ther. 2013. PMID: 23369965 Free PMC article. No abstract available.
The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis.
Nowrouzi A, Cheung WT, Li T, Zhang X, Arens A, Paruzynski A, Waddington SN, Osejindu E, Reja S, von Kalle C, Wang Y, Al-Allaf F, Gregory L, Themis M, Holder M, Dighe N, Ruthe A, Buckley SM, Bigger B, Montini E, Thrasher AJ, Andrews R, Roberts TP, Newbold RF, Coutelle C, Schmidt M, Themis M. Nowrouzi A, et al. Mol Ther. 2013 Feb;21(2):324-37. doi: 10.1038/mt.2012.224. Epub 2012 Nov 13. Mol Ther. 2013. PMID: 23299800 Free PMC article.
60 results