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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: mavilio f. 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.
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: mavilio f. 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.
The future of gene therapy.
Cavazzana-Calvo M, Thrasher A, Mavilio F. Cavazzana-Calvo M, et al. Among authors: mavilio f. Nature. 2004 Feb 26;427(6977):779-81. doi: 10.1038/427779a. Nature. 2004. PMID: 14985734 No abstract available.
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: mavilio f. 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.
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.
Charrier S, Lagresle-Peyrou C, Poletti V, Rothe M, Cédrone G, Gjata B, Mavilio F, Fischer A, Schambach A, de Villartay JP, Cavazzana M, Hacein-Bey-Abina S, Galy A. Charrier S, et al. Among authors: mavilio f. Mol Ther Methods Clin Dev. 2019 Sep 13;15:232-245. doi: 10.1016/j.omtm.2019.08.014. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31720302 Free PMC article.
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.
Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F. Poletti V, et al. Among authors: mavilio f. Mol Ther Methods Clin Dev. 2018 Nov 1;11:167-179. doi: 10.1016/j.omtm.2018.10.014. eCollection 2018 Dec 14. Mol Ther Methods Clin Dev. 2018. PMID: 30533448 Free PMC article.
Comprehensive genomic access to vector integration in clinical gene therapy.
Gabriel R, Eckenberg R, Paruzynski A, Bartholomae CC, Nowrouzi A, Arens A, Howe SJ, Recchia A, Cattoglio C, Wang W, Faber K, Schwarzwaelder K, Kirsten R, Deichmann A, Ball CR, Balaggan KS, Yáñez-Muñoz RJ, Ali RR, Gaspar HB, Biasco L, Aiuti A, Cesana D, Montini E, Naldini L, Cohen-Haguenauer O, Mavilio F, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M. Gabriel R, et al. Among authors: mavilio f. Nat Med. 2009 Dec;15(12):1431-6. doi: 10.1038/nm.2057. Epub 2009 Nov 22. Nat Med. 2009. PMID: 19966782
196 results