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345 results

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Page 1
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.
Gaspar HB, Parsley KL, Howe S, King D, Gilmour KC, Sinclair J, Brouns G, Schmidt M, Von Kalle C, Barington T, Jakobsen MA, Christensen HO, Al Ghonaium A, White HN, Smith JL, Levinsky RJ, Ali RR, Kinnon C, Thrasher AJ. Gaspar HB, et al. Among authors: von kalle c. Lancet. 2004 Dec 18-31;364(9452):2181-7. doi: 10.1016/S0140-6736(04)17590-9. Lancet. 2004. PMID: 15610804
Failure of SCID-X1 gene therapy in older patients.
Thrasher AJ, Hacein-Bey-Abina S, Gaspar HB, Blanche S, Davies EG, Parsley K, Gilmour K, King D, Howe S, Sinclair J, Hue C, Carlier F, von Kalle C, de Saint Basile G, le Deist F, Fischer A, Cavazzana-Calvo M. Thrasher AJ, et al. Among authors: von kalle c. Blood. 2005 Jun 1;105(11):4255-7. doi: 10.1182/blood-2004-12-4837. Epub 2005 Feb 1. Blood. 2005. PMID: 15687233 Free article.
Effective gene therapy with nonintegrating lentiviral vectors.
Yáñez-Muñoz RJ, Balaggan KS, MacNeil A, Howe SJ, Schmidt M, Smith AJ, Buch P, MacLaren RE, Anderson PN, Barker SE, Duran Y, Bartholomae C, von Kalle C, Heckenlively JR, Kinnon C, Ali RR, Thrasher AJ. Yáñez-Muñoz RJ, et al. Among authors: von kalle c. Nat Med. 2006 Mar;12(3):348-53. doi: 10.1038/nm1365. Epub 2006 Feb 19. Nat Med. 2006. PMID: 16491086
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: von kalle c. J Clin Invest. 2007 Aug;117(8):2241-9. doi: 10.1172/JCI31661. J Clin Invest. 2007. PMID: 17671654 Free PMC article. Clinical Trial.
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.
Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M, Kempski H, Brugman MH, Pike-Overzet K, Chatters SJ, de Ridder D, Gilmour KC, Adams S, Thornhill SI, Parsley KL, Staal FJ, Gale RE, Linch DC, Bayford J, Brown L, Quaye M, Kinnon C, Ancliff P, Webb DK, Schmidt M, von Kalle C, Gaspar HB, Thrasher AJ. Howe SJ, et al. Among authors: von kalle c. J Clin Invest. 2008 Sep;118(9):3143-50. doi: 10.1172/JCI35798. J Clin Invest. 2008. PMID: 18688286 Free PMC article. Clinical Trial.
Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice.
Themis M, Waddington SN, Schmidt M, von Kalle C, Wang Y, Al-Allaf F, Gregory LG, Nivsarkar M, Themis M, Holder MV, Buckley SM, Dighe N, Ruthe AT, Mistry A, Bigger B, Rahim A, Nguyen TH, Trono D, Thrasher AJ, Coutelle C. Themis M, et al. Among authors: von kalle c. Mol Ther. 2005 Oct;12(4):763-71. doi: 10.1016/j.ymthe.2005.07.358. Mol Ther. 2005. PMID: 16084128 Free article.
Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency.
Abdul-Razak HH, Rocca CJ, Howe SJ, Alonso-Ferrero ME, Wang J, Gabriel R, Bartholomae CC, Gan CHV, Garín MI, Roberts A, Blundell MP, Prakash V, Molina-Estevez FJ, Pantoglou J, Guenechea G, Holmes MC, Gregory PD, Kinnon C, von Kalle C, Schmidt M, Bueren JA, Thrasher AJ, Yáñez-Muñoz RJ. Abdul-Razak HH, et al. Among authors: von kalle c. Sci Rep. 2018 May 29;8(1):8214. doi: 10.1038/s41598-018-26439-9. Sci Rep. 2018. PMID: 29844458 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: von kalle c. J Clin Invest. 2007 Aug;117(8):2225-32. doi: 10.1172/JCI31659. J Clin Invest. 2007. PMID: 17671652 Free PMC article.
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: von kalle c. 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.
Hot spots of retroviral integration in human CD34+ hematopoietic cells.
Cattoglio C, Facchini G, Sartori D, Antonelli A, Miccio A, Cassani B, Schmidt M, von Kalle C, Howe S, Thrasher AJ, Aiuti A, Ferrari G, Recchia A, Mavilio F. Cattoglio C, et al. Among authors: von kalle c. Blood. 2007 Sep 15;110(6):1770-8. doi: 10.1182/blood-2007-01-068759. Epub 2007 May 16. Blood. 2007. PMID: 17507662 Free article.
345 results