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Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates.
Milani M, Annoni A, Moalli F, Liu T, Cesana D, Calabria A, Bartolaccini S, Biffi M, Russo F, Visigalli I, Raimondi A, Patarroyo-White S, Drager D, Cristofori P, Ayuso E, Montini E, Peters R, Iannacone M, Cantore A, Naldini L. Milani M, et al. Among authors: drager d. Sci Transl Med. 2019 May 22;11(493):eaav7325. doi: 10.1126/scitranslmed.aav7325. Sci Transl Med. 2019. PMID: 31118293 Free PMC article.
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates.
Milani M, Canepari C, Liu T, Biffi M, Russo F, Plati T, Curto R, Patarroyo-White S, Drager D, Visigalli I, Brombin C, Albertini P, Follenzi A, Ayuso E, Mueller C, Annoni A, Naldini L, Cantore A. Milani M, et al. Among authors: drager d. Nat Commun. 2022 May 4;13(1):2454. doi: 10.1038/s41467-022-30102-3. Nat Commun. 2022. PMID: 35508619 Free PMC article.
BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice.
Seth Chhabra E, Liu T, Kulman J, Patarroyo-White S, Yang B, Lu Q, Drager D, Moore N, Liu J, Holthaus AM, Sommer JM, Ismail A, Rabinovich D, Liu Z, van der Flier A, Goodman A, Furcht C, Tie M, Carlage T, Mauldin R, Dobrowsky TM, Liu Z, Mercury O, Zhu L, Mei B, Schellenberger V, Jiang H, Pierce GF, Salas J, Peters R. Seth Chhabra E, et al. Among authors: drager d. Blood. 2020 Apr 23;135(17):1484-1496. doi: 10.1182/blood.2019001292. Blood. 2020. PMID: 32078672 Free PMC article.
120 results