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Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates
Milani, Michela, Canepari, Cesare, Liu, Tongyao, Biffi, Mauro, Russo, Fabio, Plati, Tiziana, Curto, Rosalia, Patarroyo-White, Susannah, Drager, Douglas, Visigalli, Ilaria, Brombin, Chiara, Albertini, Paola, Follenzi, Antonia, Ayuso, Eduard, Mueller, Christian, Annoni, Andrea, Naldini, Luigi, Cantore, Alessio
Published in Nature communications (04.05.2022)
Published in Nature communications (04.05.2022)
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Journal Article
Retrieval of vector integration sites from cell-free DNA
Cesana, Daniela, Calabria, Andrea, Rudilosso, Laura, Gallina, Pierangela, Benedicenti, Fabrizio, Spinozzi, Giulio, Schiroli, Giulia, Magnani, Alessandra, Acquati, Serena, Fumagalli, Francesca, Calbi, Valeria, Witzel, Maximilian, Bushman, Frederic D., Cantore, Alessio, Genovese, Pietro, Klein, Christoph, Fischer, Alain, Cavazzana, Marina, Six, Emmanuelle, Aiuti, Alessandro, Naldini, Luigi, Montini, Eugenio
Published in Nature medicine (01.08.2021)
Published in Nature medicine (01.08.2021)
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Journal Article
Liver fibrosis negatively impacts in vivo gene transfer to murine hepatocytes
Simoni, Chiara, Nozi, Justine, Starinieri, Francesco, La Bella, Tiziana, Manta, Elisabetta, Negri, Camilla, Biffi, Mauro, Norata, Rossana, Rocchi, Martina, Sanvito, Francesca, Ronzitti, Giuseppe, Barbon, Elena, Cantore, Alessio
Published in Nature communications (10.03.2025)
Published in Nature communications (10.03.2025)
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Journal Article
Enhancing the potency of in vivo lentiviral vector mediated gene therapy to hepatocytes
Canepari, Cesare, Milani, Michela, Simoni, Chiara, Starinieri, Francesco, Volpin, Monica, Fabiano, Anna, Biffi, Mauro, Russo, Fabio, Norata, Rossana, Rocchi, Martina, Brombin, Chiara, Cugnata, Federica, Montini, Eugenio, Sanvito, Francesca, Grompe, Markus, Cantore, Alessio
Published in Nature communications (23.05.2025)
Published in Nature communications (23.05.2025)
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Journal Article
Site-specific integration and tailoring of cassette design for sustainable gene transfer
Lombardo, Angelo, Cesana, Daniela, Genovese, Pietro, Di Stefano, Bruno, Provasi, Elena, Colombo, Daniele F, Neri, Margherita, Magnani, Zulma, Cantore, Alessio, Lo Riso, Pietro, Damo, Martina, Pello, Oscar M, Holmes, Michael C, Gregory, Philip D, Gritti, Angela, Broccoli, Vania, Bonini, Chiara, Naldini, Luigi
Published in Nature methods (01.10.2011)
Published in Nature methods (01.10.2011)
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Journal Article
Identification of hepatocyte-primed cholangiocytes in the homeostatic liver by in vivo lentiviral gene transfer to mice and non-human primates
Milani, Michela, Starinieri, Francesco, Fabiano, Anna, Beretta, Stefano, Plati, Tiziana, Canepari, Cesare, Biffi, Mauro, Russo, Fabio, Berno, Valeria, Norata, Rossana, Sanvito, Francesca, Merelli, Ivan, Aloia, Luigi, Huch, Meritxell, Naldini, Luigi, Cantore, Alessio
Published in Cell reports (Cambridge) (25.03.2025)
Published in Cell reports (Cambridge) (25.03.2025)
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Journal Article
GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice
Milani, Michela, Canepari, Cesare, Assanelli, Simone, Merlin, Simone, Borroni, Ester, Starinieri, Francesco, Biffi, Mauro, Russo, Fabio, Fabiano, Anna, Zambroni, Desirèe, Annoni, Andrea, Naldini, Luigi, Follenzi, Antonia, Cantore, Alessio
Published in EMBO molecular medicine (14.06.2024)
Published in EMBO molecular medicine (14.06.2024)
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Journal Article
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
Brown, Brian D., Cantore, Alessio, Annoni, Andrea, Sergi, Lucia Sergi, Lombardo, Angelo, Della Valle, Patrizia, D'Angelo, Armando, Naldini, Luigi
Published in Blood (15.12.2007)
Published in Blood (15.12.2007)
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Journal Article
In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges
Cantore, Alessio, Fraldi, Alessandro, Meneghini, Vasco, Gritti, Angela
Published in Frontiers in medicine (18.01.2022)
Published in Frontiers in medicine (18.01.2022)
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Journal Article
Genome editing for scalable production of alloantigen‐free lentiviral vectors for in vivo gene therapy
Milani, Michela, Annoni, Andrea, Bartolaccini, Sara, Biffi, Mauro, Russo, Fabio, Di Tomaso, Tiziano, Raimondi, Andrea, Lengler, Johannes, Holmes, Michael C, Scheiflinger, Friedrich, Lombardo, Angelo, Cantore, Alessio, Naldini, Luigi
Published in EMBO molecular medicine (01.11.2017)
Published in EMBO molecular medicine (01.11.2017)
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Journal Article
Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering
Soldi, Monica, Sergi Sergi, Lucia, Unali, Giulia, Kerzel, Thomas, Cuccovillo, Ivan, Capasso, Paola, Annoni, Andrea, Biffi, Mauro, Rancoita, Paola Maria Vittoria, Cantore, Alessio, Lombardo, Angelo, Naldini, Luigi, Squadrito, Mario Leonardo, Kajaste-Rudnitski, Anna
Published in Molecular therapy. Methods & clinical development (11.12.2020)
Published in Molecular therapy. Methods & clinical development (11.12.2020)
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Journal Article
Liver gene therapy by lentiviral vectors reverses anti‐factor IX pre‐existing immunity in haemophilic mice
Annoni, Andrea, Cantore, Alessio, Della Valle, Patrizia, Goudy, Kevin, Akbarpour, Mahzad, Russo, Fabio, Bartolaccini, Sara, D'Angelo, Armando, Roncarolo, Maria Grazia, Naldini, Luigi
Published in EMBO molecular medicine (01.11.2013)
Published in EMBO molecular medicine (01.11.2013)
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Journal Article
Modulation of immune responses in lentiviral vector-mediated gene transfer
Annoni, Andrea, Gregori, Silvia, Naldini, Luigi, Cantore, Alessio
Published in Cellular immunology (01.08.2019)
Published in Cellular immunology (01.08.2019)
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Journal Article
Low-density lipoproteins receptor (LDLR) liver gene therapy normalizes LDL cholesterol (LDL-C) and prevents atherosclerosis in a familial hypercholesterolemia (FH) mouse model
Canepari, Cesare, Milani, Michela, Starinieri, Francesco, Fabiano, Anna, Russo, Fabio, Norata, Rossana, Rocchi, Martina, Biffi, Mauro, Sanvito, Francesca, Cantore, Alessio
Published in Atherosclerosis (01.08.2024)
Published in Atherosclerosis (01.08.2024)
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Journal Article