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Immune tolerance promotion by LSEC-specific lentiviral vector-mediated expression of the transgene regulated by the stabilin-2 promoter 1-gen-2024 Borroni, Ester; Borsotti, Chiara; Cirsmaru, Roberta Annamaria; Kalandadze, Vakhtang; Famà, Rosella; Merlin, Simone; Brown, Brian; Follenzi, Antonia
Therapeutic potential of fetal liver cells transplantation in hemophilia A mice 1-gen-2023 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
Liver gene therapy with intein-mediated F8 trans-splicing corrects mouse haemophilia A 1-gen-2022 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
Therapeutic correction of hemophilia A by transplantation of hPSC-derived liver sinusoidal endothelial cell progenitors (Cell reports (2022) 39 1 (110621) PII: S2211-1247(22)00523-X) 1-gen-2022 Gage, B. K.; Merlin, S.; Olgasi, C.; Follenzi, A.; Keller, G. M.
Factor VIII as a potential player in cancer pathophysiology 1-gen-2021 Walker, Gillian E.; Merlin, Simone; Zanolini, Diego; Vandoni, Andrea; Volpe, Alessandro; Gaidano, Gianluca; Valente, Guido; Olivero, Martina; Follenzi, Antonia
Dissection of pleiotropic effects of variants in and adjacent to F8 exon 19 and rescue of mRNA splicing and protein function 1-gen-2021 Lombardi, S.; Leo, G.; Merlin, S.; Follenzi, A.; Mcvey, J. H.; Maestri, I.; Bernardi, F.; Pinotti, M.; Balestra, D.
Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device 1-gen-2021 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
Deciphering the Ets-1/2-mediated transcriptional regulation of F8 gene identifies a minimal F8 promoter for hemophilia A gene therapy 1-gen-2021 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
Identification and functional characterization of a novel splicing variant in the F8 coagulation gene causing severe hemophilia A 1-gen-2020 Famà, Rosella; Borroni, Ester; Zanolini, Diego; Merlin, Simone; Bruscaggin, Valentina; Walker, Gillian E; Olgasi, Cristina; Babu, Deepak; Agnelli Giacchello, Jacopo; Valeri, Federica; Giordano, Mara; Borchiellini, Alessandra; Follenzi, Antonia
Thyroid hormone inhibits hepatocellular carcinoma progression via induction of differentiation and metabolic reprogramming 1-gen-2020 Kowalik, M. A.; Puliga, E.; Cabras, L.; Sulas, P.; Petrelli, A.; Perra, A.; Ledda-Columbano, G. M.; Morandi, A.; Merlin, S.; Orru, C.; Sanchez-Martin, C.; Fornari, F.; Gramantieri, L.; Parri, M.; Rasola, A.; Bellomo, S. E.; Sebastian, C.; Follenzi, A.; Giordano, S.; Columbano, A.
Escape or Fight: Inhibitors in Hemophilia A 1-gen-2020 Merlin, S.; Follenzi, A.
Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells 1-gen-2020 Bittorf, P.; Bergmann, T.; Merlin, S.; Olgasi, C.; Pullig, O.; Sanzenbacher, R.; Zierau, M.; Walles, H.; Follenzi, A.; Braspenning, J.
FVIII expression by its native promoter sustains long-term correction avoiding immune response in hemophilic mice 1-gen-2019 Merlin, Simone; Famà, Rosella; Borroni, Ester; Zanolini, Diego; Bruscaggin, Valentina; Zucchelli, Silvia; Follenzi, Antonia
Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors 1-gen-2019 Merlin, Simone; Follenzi, Antonia
Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A 1-gen-2018 Olgasi, C; Talmon, M; Merlin, S; Cucci, Alessia; Richaud-Patin, Y; Ranaldo, G; Colangelo, D; Di Scipio, F; Berta, Gn; Borsotti, C; Valeri, F; Faraldi, F; Prat, M; Messina, M; Schinco, P; Lombardo, A; Raya, A; Follenzi, A.
A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice 1-gen-2017 Merlin, Simone; Cannizzo, ELVIRA STEFANIA; Borroni, Ester; Bruscaggin, Valentina; Schinco, Piercarla; Tulalamba, Warut; Chuah, Marinee K.; Arruda, Valder R.; Vandendriessche, Thierry; Prat, Maria Giovanna; Valente, Guido; Follenzi, Antonia
A long term, non-tumorigenic rat hepatocyte cell line and its malignant counterpart, as tools to study hepatocarcinogenesis 1-gen-2017 Angioni, Maria Maddalena; Bellofatto, Kevin; Merlin, Simone; Menegon, Silvia; Perra, Andrea; Petrelli, Annalisa; Sulas, Pia; Giordano, Silvia; Columbano, Amedeo; Follenzi, Antonia
The Dendritic Cell Major Histocompatibility Complex II (MHC II) Peptidome Derives from a Variety of Processing Pathways and Includes Peptides with a Broad Spectrum of HLA-DM Sensitivity 1-gen-2016 Clement, Cristina C; Becerra, Aniuska; Yin, Liusong; Zolla, Valerio; Huang, Liling; Merlin, Simone; Follenzi, Antonia; Shaffer, Scott A; Stern, Lawrence J; Santambrogio, Laura
Kupffer cell transplantation in mice for elucidating monocyte/macrophage biology and for potential in cell or gene therapy 1-gen-2016 Merlin, Simone; Bhargava, Kk; Ranaldo, G; Zanolini, D; Palestro, Cj; Santambrogio, L; Prat, Maria Giovanna; Follenzi, Antonia; Gupta, S.
Activity and High-Order Effective Connectivity Alterations in Sanfilippo C Patient-Specific Neuronal Networks 1-gen-2015 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
Mostrati risultati da 1 a 20 di 37
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