Escape or Fight: Inhibitors in Hemophilia A von Merlin, Simone, Follenzi, Antonia

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Factor VIII promotes angiogenesis and vessel stability regulating extracellular matrix proteins von Olgasi, Cristina, Cucci, Alessia, Molineris, Ivan, Assanelli, Simone, Anselmi, Francesca, Borsotti, Chiara, Sgromo, Chiara, Lauria, Andrea, Merlin, Simone, Walker, Gillian E, Oliviero, Salvatore, Follenzi, Antonia

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Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors von Merlin, Simone, Follenzi, Antonia

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Therapeutic correction of hemophilia A by transplantation of hPSC-derived liver sinusoidal endothelial cell progenitors von Gage, Blair K., Merlin, Simone, Olgasi, Cristina, Follenzi, Antonia, Keller, Gordon M.

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GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice von 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

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Role of bone marrow transplantation for correcting hemophilia A in mice von Follenzi, Antonia, Raut, Sanj, Merlin, Simone, Sarkar, Rita, Gupta, Sanjeev

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Therapeutic potential of fetal liver cell transplantation in hemophilia A mice von Merlin, Simone, Akula, Saicharan, Cottonaro, Alessia, Garcia-Leal, Tamara, Serrano, Luis Javier, Borroni, Ester, Kalandadze, Vakhtang, Galiano, Rocio, Borsotti, Chiara, Liras, Antonio, Sanchez, María José, Follenzi, Antonia

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Immune tolerance promotion by LSEC-specific lentiviral vector-mediated expression of the transgene regulated by the stabilin-2 promoter von Borroni, Ester, Borsotti, Chiara, Cirsmaru, Roberta A., Kalandadze, Vakhtang, Famà, Rosella, Merlin, Simone, Brown, Brian, Follenzi, Antonia

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Deciphering the Ets-1/2-mediated transcriptional regulation of F8 gene identifies a minimal F8 promoter for hemophilia A gene therapy von Fama, Rosella, Borroni, Ester, Merlin, Simone, Airoldi, Chiara, Pignani, Silvia, Cucci, Alessia, Cora, Davide, Bruscaggin, Valentina, Scardellato, Sharon, Faletti, Stefania, Pelicci, Giuliana, Pinotti, Mirko, Walker, Gillian E., Follenzi, Antonia

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Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A von Esposito, Federica, Lyubenova, Hristiana, Tornabene, Patrizia, Auricchio, Stefano, Iuliano, Antonella, Nusco, Edoardo, Merlin, Simone, Olgasi, Cristina, Manni, Giorgia, Gargaro, Marco, Fallarino, Francesca, Follenzi, Antonia, Auricchio, Alberto

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Extrahepatic sources of factor VIII potentially contribute to the coagulation cascade correcting the bleeding phenotype of mice with hemophilia A von Zanolini, Diego, Merlin, Simone, Feola, Maria, Ranaldo, Gabriella, Amoruso, Angela, Gaidano, Gianluca, Zaffaroni, Mauro, Ferrero, Alessandro, Brunelleschi, Sandra, Valente, Guido, Gupta, Sanjeev, Prat, Maria, Follenzi, Antonia

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Human Cardiac Progenitor Spheroids Exhibit Enhanced Engraftment Potential von Oltolina, Francesca, Zamperone, Andrea, Colangelo, Donato, Gregoletto, Luca, Reano, Simone, Pietronave, Stefano, Merlin, Simone, Talmon, Maria, Novelli, Eugenio, Diena, Marco, Nicoletti, Carmine, Musarò, Antonio, Filigheddu, Nicoletta, Follenzi, Antonia, Prat, Maria

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Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A von Olgasi, Cristina, Talmon, Maria, Merlin, Simone, Cucci, Alessia, Richaud-Patin, Yvonne, Ranaldo, Gabriella, Colangelo, Donato, Di Scipio, Federica, Berta, Giovanni N., Borsotti, Chiara, Valeri, Federica, Faraldi, Francesco, Prat, Maria, Messina, Maria, Schinco, Piercarla, Lombardo, Angelo, Raya, Angel, Follenzi, Antonia

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Kupffer Cell Transplantation in Mice for Elucidating Monocyte/Macrophage Biology and for Potential in Cell or Gene Therapy von Merlin, Simone, Bhargava, Kuldeep K, Ranaldo, Gabriella, Zanolini, Diego, Palestro, Christopher J, Santambrogio, Laura, Prat, Maria, Follenzi, Antonia, Gupta, Sanjeev

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Diacylglycerol kinases are essential for hepatocyte growth factor‐dependent proliferation and motility of Kaposi’s sarcoma cells von Baldanzi, Gianluca, Pietronave, Stefano, Locarno, Deborah, Merlin, Simone, Porporato, Paolo, Chianale, Federica, Filigheddu, Nicoletta, Cantelmo, Anna Rita, Albini, Adriana, Graziani, Andrea, Prat, Maria

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Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device von Olgasi, Cristina, Borsotti, Chiara, Merlin, Simone, Bergmann, Thorsten, Bittorf, Patrick, Adewoye, Adeolu Badi, Wragg, Nicholas, Patterson, Kelcey, Calabria, Andrea, Benedicenti, Fabrizio, Cucci, Alessia, Borchiellini, Alessandra, Pollio, Berardino, Montini, Eugenio, Mazzuca, Delfina M., Zierau, Martin, Stolzing, Alexandra, Toleikis, Philip.M., Braspenning, Joris, Follenzi, Antonia

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Dissecting the transcriptional phenotype of ribosomal protein deficiency: implications for Diamond-Blackfan Anemia von Aspesi, Anna, Pavesi, Elisa, Robotti, Elisa, Crescitelli, Rossella, Boria, Ilenia, Avondo, Federica, Moniz, Hélène, Da Costa, Lydie, Mohandas, Narla, Roncaglia, Paola, Ramenghi, Ugo, Ronchi, Antonella, Gustincich, Stefano, Merlin, Simone, Marengo, Emilio, Ellis, Steven R., Follenzi, Antonia, Santoro, Claudio, Dianzani, Irma

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Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells von Bittorf, Patrick, Bergmann, Thorsten, Merlin, Simone, Olgasi, Cristina, Pullig, Oliver, Sanzenbacher, Ralf, Zierau, Martin, Walles, Heike, Follenzi, Antonia, Braspenning, Joris

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Deletion of the ectodomain unleashes the transforming, invasive, and tumorigenic potential of the MET oncogene von Merlin, Simone, Pietronave, Stefano, Locarno, Deborah, Valente, Guido, Follenzi, Antonia, Prat, Maria

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Activity and High-Order Effective Connectivity Alterations in Sanfilippo C Patient-Specific Neuronal Networks von Canals, Isaac, Soriano, Jordi, Orlandi, Javier G., Torrent, Roger, Richaud-Patin, Yvonne, Jiménez-Delgado, Senda, Merlin, Simone, Follenzi, Antonia, Consiglio, Antonella, Vilageliu, Lluïsa, Grinberg, Daniel, Raya, Angel

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