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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. Sci Transl Med. 2015 Mar 4;7(277):277ra28. doi: 10.1126/scitranslmed.aaa1405. Sci Transl Med. 2015. PMID: 25739762 Free PMC article.
Immune responses in liver-directed lentiviral gene therapy.
Annoni A, Goudy K, Akbarpour M, Naldini L, Roncarolo MG. Annoni A, et al. Transl Res. 2013 Apr;161(4):230-40. doi: 10.1016/j.trsl.2012.12.018. Epub 2013 Jan 26. Transl Res. 2013. PMID: 23360745 Review.
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. Hepatology. 2011 May;53(5):1696-707. doi: 10.1002/hep.24230. Hepatology. 2011. PMID: 21520180 Free PMC article.