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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: bartholomae cc. Sci Transl Med. 2015 Mar 4;7(277):277ra28. doi: 10.1126/scitranslmed.aaa1405. Sci Transl Med. 2015. PMID: 25739762 Free PMC article.
Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer.
Ranzani M, Cesana D, Bartholomae CC, Sanvito F, Pala M, Benedicenti F, Gallina P, Sergi LS, Merella S, Bulfone A, Doglioni C, von Kalle C, Kim YJ, Schmidt M, Tonon G, Naldini L, Montini E. Ranzani M, et al. Among authors: bartholomae cc. Nat Methods. 2013 Feb;10(2):155-61. doi: 10.1038/nmeth.2331. Epub 2013 Jan 13. Nat Methods. 2013. PMID: 23314173 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. 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.
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: bartholomae cc. 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.
Lentiviral vector integration profiles differ in rodent postmitotic tissues.
Bartholomae CC, Arens A, Balaggan KS, Yáñez-Muñoz RJ, Montini E, Howe SJ, Paruzynski A, Korn B, Appelt JU, Macneil A, Cesana D, Abel U, Glimm H, Naldini L, Ali RR, Thrasher AJ, von Kalle C, Schmidt M. Bartholomae CC, et al. Mol Ther. 2011 Apr;19(4):703-10. doi: 10.1038/mt.2011.19. Epub 2011 Mar 1. Mol Ther. 2011. PMID: 21364536 Free PMC article.
Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk.
Mátrai J, Cantore A, Bartholomae CC, Annoni A, Wang W, Acosta-Sanchez A, Samara-Kuko E, De Waele L, Ma L, Genovese P, Damo M, Arens A, Goudy K, Nichols TC, von Kalle C, L Chuah MK, Roncarolo MG, Schmidt M, Vandendriessche T, Naldini L. Mátrai J, et al. Among authors: bartholomae cc. Hepatology. 2011 May;53(5):1696-707. doi: 10.1002/hep.24230. Hepatology. 2011. PMID: 21520180 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: bartholomae cc. 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.
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: bartholomae cc. Nat Med. 2009 Dec;15(12):1431-6. doi: 10.1038/nm.2057. Epub 2009 Nov 22. Nat Med. 2009. PMID: 19966782
44 results