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Gene therapy of primary T cell immunodeficiencies.
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Fischer A, et al. Among authors: cavazzana calvo m. Gene. 2013 Aug 10;525(2):170-3. doi: 10.1016/j.gene.2013.03.092. Epub 2013 Apr 10. Gene. 2013. PMID: 23583799 Free article. Review.
Critical variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency.
van der Loo JC, Swaney WP, Grassman E, Terwilliger A, Higashimoto T, Schambach A, Hacein-Bey-Abina S, Nordling DL, Cavazzana-Calvo M, Thrasher AJ, Williams DA, Reeves L, Malik P. van der Loo JC, et al. Gene Ther. 2012 Aug;19(8):872-6. doi: 10.1038/gt.2012.37. Epub 2012 May 3. Gene Ther. 2012. PMID: 22551777 Free PMC article.
Gene therapy for primary adaptive immune deficiencies.
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Fischer A, et al. J Allergy Clin Immunol. 2011 Jun;127(6):1356-9. doi: 10.1016/j.jaci.2011.04.030. J Allergy Clin Immunol. 2011. PMID: 21624615 Review.
Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.
Tolar J, Adair JE, Antoniou M, Bartholomae CC, Becker PS, Blazar BR, Bueren J, Carroll T, Cavazzana-Calvo M, Clapp DW, Dalgleish R, Galy A, Gaspar HB, Hanenberg H, Von Kalle C, Kiem HP, Lindeman D, Naldini L, Navarro S, Renella R, Rio P, Sevilla J, Schmidt M, Verhoeyen E, Wagner JE, Williams DA, Thrasher AJ. Tolar J, et al. Mol Ther. 2011 Jul;19(7):1193-8. doi: 10.1038/mt.2011.78. Epub 2011 May 3. Mol Ther. 2011. PMID: 21540837 Free PMC article. Review.
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. 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.
199 results