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Liver-directed lentiviral gene therapy in a dog model of hemophilia B.
Cantore A, Ranzani M, Bartholomae CC, Volpin M, Valle PD, Sanvito F, Sergi LS, Gallina P, Benedicenti F, Bellinger D, Raymer R, Merricks E, Bellintani F, Martin S, Doglioni C, D'Angelo A, VandenDriessche T, Chuah MK, Schmidt M, Nichols T, Montini E, Naldini L. Cantore A, et al. Among authors: ranzani m. Sci Transl Med. 2015 Mar 4;7(277):277ra28. doi: 10.1126/scitranslmed.aaa1405. Sci Transl Med. 2015. PMID: 25739762 Free PMC article.
The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy.
Montini E, Cesana D, Schmidt M, Sanvito F, Bartholomae CC, Ranzani M, Benedicenti F, Sergi LS, Ambrosi A, Ponzoni M, Doglioni C, Di Serio C, von Kalle C, Naldini L. Montini E, et al. Among authors: ranzani m. J Clin Invest. 2009 Apr;119(4):964-75. doi: 10.1172/JCI37630. Epub 2009 Mar 23. J Clin Invest. 2009. PMID: 19307726 Free PMC article.
Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo.
Cesana D, Ranzani M, Volpin M, Bartholomae C, Duros C, Artus A, Merella S, Benedicenti F, Sergi Sergi L, Sanvito F, Brombin C, Nonis A, Serio CD, Doglioni C, von Kalle C, Schmidt M, Cohen-Haguenauer O, Naldini L, Montini E. Cesana D, et al. Among authors: ranzani m. Mol Ther. 2014 Apr;22(4):774-85. doi: 10.1038/mt.2014.3. Epub 2014 Jan 20. Mol Ther. 2014. PMID: 24441399 Free PMC article.
Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome.
Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, Bosticardo M, Schmidt M, von Kalle C, Montini E, Biffi A, Roncarolo MG, Naldini L, Villa A, Aiuti A. Scaramuzza S, et al. Among authors: ranzani m. Mol Ther. 2013 Jan;21(1):175-84. doi: 10.1038/mt.2012.23. Epub 2012 Feb 28. Mol Ther. 2013. PMID: 22371846 Free PMC article.
Cancer gene discovery: exploiting insertional mutagenesis.
Ranzani M, Annunziato S, Adams DJ, Montini E. Ranzani M, et al. Mol Cancer Res. 2013 Oct;11(10):1141-58. doi: 10.1158/1541-7786.MCR-13-0244. Epub 2013 Aug 8. Mol Cancer Res. 2013. PMID: 23928056 Free PMC article. Review.
Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection.
Biffi A, Bartolomae CC, Cesana D, Cartier N, Aubourg P, Ranzani M, Cesani M, Benedicenti F, Plati T, Rubagotti E, Merella S, Capotondo A, Sgualdino J, Zanetti G, von Kalle C, Schmidt M, Naldini L, Montini E. Biffi A, et al. Among authors: ranzani m. Blood. 2011 May 19;117(20):5332-9. doi: 10.1182/blood-2010-09-306761. Epub 2011 Mar 14. Blood. 2011. PMID: 21403130 Free article.
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