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Page 1
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: brugman mh. 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.
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: brugman mh. J Clin Invest. 2007 Aug;117(8):2225-32. doi: 10.1172/JCI31659. J Clin Invest. 2007. PMID: 17671652 Free PMC article.
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo.
Schwarzwaelder K, Howe SJ, Schmidt M, Brugman MH, Deichmann A, Glimm H, Schmidt S, Prinz C, Wissler M, King DJ, Zhang F, Parsley KL, Gilmour KC, Sinclair J, Bayford J, Peraj R, Pike-Overzet K, Staal FJ, de Ridder D, Kinnon C, Abel U, Wagemaker G, Gaspar HB, Thrasher AJ, von Kalle C. Schwarzwaelder K, et al. Among authors: brugman mh. J Clin Invest. 2007 Aug;117(8):2241-9. doi: 10.1172/JCI31661. J Clin Invest. 2007. PMID: 17671654 Free PMC article. Clinical Trial.
Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.
Huston MW, van Til NP, Visser TP, Arshad S, Brugman MH, Cattoglio C, Nowrouzi A, Li Y, Schambach A, Schmidt M, Baum C, von Kalle C, Mavilio F, Zhang F, Blundell MP, Thrasher AJ, Verstegen MM, Wagemaker G. Huston MW, et al. Among authors: brugman mh. Mol Ther. 2011 Oct;19(10):1867-77. doi: 10.1038/mt.2011.127. Epub 2011 Jul 12. Mol Ther. 2011. PMID: 21750532 Free PMC article.
The genomic risk of somatic gene therapy.
Kustikova O, Brugman M, Baum C. Kustikova O, et al. Semin Cancer Biol. 2010 Aug;20(4):269-78. doi: 10.1016/j.semcancer.2010.06.003. Epub 2010 Jul 1. Semin Cancer Biol. 2010. PMID: 20600920 Review.
CD133-targeted gene transfer into long-term repopulating hematopoietic stem cells.
Brendel C, Goebel B, Daniela A, Brugman M, Kneissl S, Schwäble J, Kaufmann KB, Müller-Kuller U, Kunkel H, Chen-Wichmann L, Abel T, Serve H, Bystrykh L, Buchholz CJ, Grez M. Brendel C, et al. Mol Ther. 2015 Jan;23(1):63-70. doi: 10.1038/mt.2014.173. Epub 2014 Sep 5. Mol Ther. 2015. PMID: 25189742 Free PMC article.
55 results