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Successful Combination of Sequential Gene Therapy and Rescue Allo-HSCT in Two Children with X-CGD - Importance of Timing.
Siler U, Paruzynski A, Holtgreve-Grez H, Kuzmenko E, Koehl U, Renner ED, Alhan C, de Loosdrecht AA, Schwäble J, Pfluger T, Tchinda J, Schmugge M, Jauch A, Naundorf S, Kühlcke K, Notheis G, Güngor T, Kalle CV, Schmidt M, Grez M, Seger R, Reichenbach J. Siler U, et al. Among authors: paruzynski a. Curr Gene Ther. 2015;15(4):416-27. doi: 10.2174/1566523215666150515145255. Curr Gene Ther. 2015. PMID: 25981636 Clinical Trial.
Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy.
Deichmann A, Brugman MH, Bartholomae CC, Schwarzwaelder K, Verstegen MM, Howe SJ, Arens A, Ott MG, Hoelzer D, Seger R, Grez M, Hacein-Bey-Abina S, Cavazzana-Calvo M, Fischer A, Paruzynski A, Gabriel R, Glimm H, Abel U, Cattoglio C, Mavilio F, Cassani B, Aiuti A, Dunbar CE, Baum C, Gaspar HB, Thrasher AJ, von Kalle C, Schmidt M, Wagemaker G. Deichmann A, et al. Among authors: paruzynski a. Mol Ther. 2011 Nov;19(11):2031-9. doi: 10.1038/mt.2011.178. Epub 2011 Aug 23. Mol Ther. 2011. PMID: 21862999 Free PMC article.
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
Braun CJ, Boztug K, Paruzynski A, Witzel M, Schwarzer A, Rothe M, Modlich U, Beier R, Göhring G, Steinemann D, Fronza R, Ball CR, Haemmerle R, Naundorf S, Kühlcke K, Rose M, Fraser C, Mathias L, Ferrari R, Abboud MR, Al-Herz W, Kondratenko I, Maródi L, Glimm H, Schlegelberger B, Schambach A, Albert MH, Schmidt M, von Kalle C, Klein C. Braun CJ, et al. Among authors: paruzynski a. Sci Transl Med. 2014 Mar 12;6(227):227ra33. doi: 10.1126/scitranslmed.3007280. Sci Transl Med. 2014. PMID: 24622513 Clinical Trial.
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. Among authors: paruzynski a. 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.
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