Hematopoietic Cells from Pluripotent Stem Cells: Hope and Promise for the Treatment of Inherited Blood Disorders

por Rao, Ilaria, Crisafulli, Laura, Paulis, Marianna, Ficara, Francesca
Publicado 2022

A pre-screening FISH-based method to detect CRISPR/Cas9 off-targets in mouse embryonic stem cells

por Paulis, Marianna, Castelli, Alessandra, Lizier, Michela, Susani, Lucia, Lucchini, Franco, Villa, Anna, Vezzoni, Paolo
Publicado 2015

Fusion between cancer cells and macrophages occurs in a murine model of spontaneous neu(+) breast cancer without increasing its metastatic potential

por Lizier, Michela, Anselmo, Achille, Mantero, Stefano, Ficara, Francesca, Paulis, Marianna, Vezzoni, Paolo, Lucchini, Franco, Pacchiana, Giovanni
Publicado 2016

Where Are We with RPE Replacement Therapy? A Translational Review from the Ophthalmologist Perspective

por Raimondi, Raffaele, Zollet, Piero, De Rosa, Francesco Paolo, Tsoutsanis, Panagiotis, Stravalaci, Matteo, Paulis, Marianna, Inforzato, Antonio, Romano, Mario R.
Publicado 2022

P1405: IN-DEPTH GENOME INTEGRITY EVALUATION AND GMP-COMPLIANT MANUFACTURING OF HDR GENE EDITED CD4+ T CELLS FOR THE TREATMENT OF HYPER IGM 1

por Canarutto, Daniele, Asperti, Claudia, Vavassori, Valentina, Porcellini, Simona, Rovelli, Elisabetta, Paulis, Marianna, Ferrari, Samuele, Plati, Tiziana, Sergi Sergi, Lucia, Villa, Anna, Radrizzani, Marina, Naldini, Luigi
Publicado 2023

Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders

por Paulis, Marianna, Susani, Lucia, Castelli, Alessandra, Suzuki, Teruhiko, Hara, Takahiko, Straniero, Letizia, Duga, Stefano, Strina, Dario, Mantero, Stefano, Caldana, Elena, Sergi, Lucia Sergi, Villa, Anna, Vezzoni, Paolo
Publicado 2020

Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient- and cell type-specific disease phenotypes

por Mangiameli, Elisabeth, Cecchele, Anna, Morena, Francesco, Sanvito, Francesca, Matafora, Vittoria, Cattaneo, Angela, della Volpe, Lucrezia, Gnani, Daniela, Paulis, Marianna, Susani, Lucia, Martino, Sabata, Di Micco, Raffaella, Bachi, Angela, Gritti, Angela
Publicado 2021

Chromosome transplantation as a novel approach for correcting complex genomic disorders

por Paulis, Marianna, Castelli, Alessandra, Susani, Lucia, Lizier, Michela, Lagutina, Irina, Focarelli, Maria Luisa, Recordati, Camilla, Uva, Paolo, Faggioli, Francesca, Neri, Tui, Scanziani, Eugenio, Galli, Cesare, Lucchini, Franco, Villa, Anna, Vezzoni, Paolo
Publicado 2015

Generation of Human Induced Pluripotent Stem Cell‐Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy

por Meneghini, Vasco, Frati, Giacomo, Sala, Davide, De Cicco, Silvia, Luciani, Marco, Cavazzin, Chiara, Paulis, Marianna, Mentzen, Wieslawa, Morena, Francesco, Giannelli, Serena, Sanvito, Francesca, Villa, Anna, Bulfone, Alessandro, Broccoli, Vania, Martino, Sabata, Gritti, Angela
Publicado 2016

The K219T-Lamin mutation induces conduction defects through epigenetic inhibition of SCN5A in human cardiac laminopathy

por Salvarani, Nicolò, Crasto, Silvia, Miragoli, Michele, Bertero, Alessandro, Paulis, Marianna, Kunderfranco, Paolo, Serio, Simone, Forni, Alberto, Lucarelli, Carla, Dal Ferro, Matteo, Larcher, Veronica, Sinagra, Gianfranco, Vezzoni, Paolo, Murry, Charles E., Faggian, Giuseppe, Condorelli, Gianluigi, Di Pasquale, Elisa
Publicado 2019

Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4(+) T‐cells for the treatment of Hyper IgM1

por Canarutto, Daniele, Asperti, Claudia, Vavassori, Valentina, Porcellini, Simona, Rovelli, Elisabetta, Paulis, Marianna, Ferrari, Samuele, Varesi, Angelica, Fiumara, Martina, Jacob, Aurelien, Sergi Sergi, Lucia, Visigalli, Ilaria, Ferrua, Francesca, González‐Granado, Luis Ignacio, Lougaris, Vassilios, Finocchi, Andrea, Villa, Anna, Radrizzani, Marina, Naldini, Luigi
Publicado 2023

Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts

por Neri, Tui, Muggeo, Sharon, Paulis, Marianna, Caldana, Maria Elena, Crisafulli, Laura, Strina, Dario, Focarelli, Maria Luisa, Faggioli, Francesca, Recordati, Camilla, Scaramuzza, Samantha, Scanziani, Eugenio, Mantero, Stefano, Buracchi, Chiara, Sobacchi, Cristina, Lombardo, Angelo, Naldini, Luigi, Vezzoni, Paolo, Villa, Anna, Ficara, Francesca
Publicado 2015

Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons

por Mazzara, Pietro Giuseppe, Muggeo, Sharon, Luoni, Mirko, Massimino, Luca, Zaghi, Mattia, Valverde, Parisa Tajalli-Tehrani, Brusco, Simone, Marzi, Matteo Jacopo, Palma, Cecilia, Colasante, Gaia, Iannielli, Angelo, Paulis, Marianna, Cordiglieri, Chiara, Giannelli, Serena Gea, Podini, Paola, Gellera, Cinzia, Taroni, Franco, Nicassio, Francesco, Rasponi, Marco, Broccoli, Vania
Publicado 2020

Dysfunctional Extracellular Matrix Remodeling Supports Perianal Fistulizing Crohn′s Disease by a Mechanoregulated Activation of the Epithelial-to-Mesenchymal Transition

por Rizzo, Giulia, Rubbino, Federica, Elangovan, Sudharshan, Sammarco, Giusy, Lovisa, Sara, Restelli, Silvia, Pineda Chavez, Samuel Elias, Massimino, Luca, Lamparelli, Luigi, Paulis, Marianna, Maroli, Annalisa, Roda, Giulia, Shalaby, Mohammad, Carvello, Michele, Foppa, Caterina, Drummond, Sheona P., Spaggiari, Paola, Ungaro, Federica, Spinelli, Antonino, Malesci, Alberto, Repici, Alessandro, Day, Anthony J., Armuzzi, Alessandro, Danese, Silvio, Vetrano, Stefania
Publicado 2022