Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors

por Merlin, Simone, Follenzi, Antonia
Publicado 2019

Escape or Fight: Inhibitors in Hemophilia A

por Merlin, Simone, Follenzi, Antonia
Publicado 2020

Pharmacological Preconditioning by Adenosine A2a Receptor Stimulation: Features of the Protected Liver Cell Phenotype

por Alchera, Elisa, Imarisio, Chiara, Mandili, Giorgia, Merlin, Simone, Chandrashekar, Bangalore R., Novelli, Francesco, Follenzi, Antonia, Carini, Rita
Publicado 2015

Factor VIII as a potential player in cancer pathophysiology

por Walker, Gillian E., Merlin, Simone, Zanolini, Diego, Vandoni, Andrea, Volpe, Alessandro, Gaidano, Gianluca, Valente, Guido, Olivero, Martina, Follenzi, Antonia
Publicado 2022

Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells

por Bittorf, Patrick, Bergmann, Thorsten, Merlin, Simone, Olgasi, Cristina, Pullig, Oliver, Sanzenbacher, Ralf, Zierau, Martin, Walles, Heike, Follenzi, Antonia, Braspenning, Joris
Publicado 2020

A long term, non-tumorigenic rat hepatocyte cell line and its malignant counterpart, as tools to study hepatocarcinogenesis

por Angioni, Maria Maddalena, Bellofatto, Kevin, Merlin, Simone, Menegon, Silvia, Perra, Andrea, Petrelli, Annalisa, Sulas, Pia, Giordano, Silvia, Columbano, Amedeo, Follenzi, Antonia
Publicado 2017

Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A

por 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
Publicado 2022

Therapeutic potential of fetal liver cell transplantation in hemophilia A mice

por 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
Publicado 2023

Activity and High-Order Effective Connectivity Alterations in Sanfilippo C Patient-Specific Neuronal Networks

por 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
Publicado 2015

Deciphering the Ets-1/2-mediated transcriptional regulation of F8 gene identifies a minimal F8 promoter for hemophilia A gene therapy

por Famà, Rosella, Borroni, Ester, Merlin, Simone, Airoldi, Chiara, Pignani, Silvia, Cucci, Alessia, Corà, Davide, Bruscaggin, Valentina, Scardellato, Sharon, Faletti, Stefania, Pelicci, Giuliana, Pinotti, Mirko, Walker, Gillian E., Follenzi, Antonia
Publicado 2020

Human Cardiac Progenitor Spheroids Exhibit Enhanced Engraftment Potential

por 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
Publicado 2015

Correction: Human Cardiac Progenitor Spheroids Exhibit Enhanced Engraftment Potential

por 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
Publicado 2015

Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A

por 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
Publicado 2018

Dissecting the transcriptional phenotype of ribosomal protein deficiency: implications for Diamond-Blackfan Anemia

por 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
Publicado 2014

Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device

por 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
Publicado 2021