Hematopoietic Stem Cell Transplantation for Combined Immunodeficiencies, on Behalf of IEWP-EBMT

Tekijä Neven, Benedicte, Ferrua, Francesca
Julkaisussa Front Pediatr (2020)

Gene Therapy for Wiskott-Aldrich Syndrome: History, New Vectors, Future Directions

Tekijä Ferrua, Francesca, Marangoni, Francesco, Aiuti, Alessandro, Roncarolo, Maria Grazia
Julkaisussa J Allergy Clin Immunol (2020)

Role of reduced intensity conditioning in T-cell and B-cell immune reconstitution after HLA-identical bone marrow transplantation in ADA-SCID

Tekijä Cancrini, Caterina, Ferrua, Francesca, Scarselli, Alessia, Brigida, Immacolata, Romiti, Maria Luisa, Barera, Graziano, Finocchi, Andrea, Roncarolo, Maria Grazia, Caniglia, Maurizio, Aiuti, Alessandro
Julkaistu 2010

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

Tekijä Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Roncarolo, Maria Grazia, Aiuti, Alessandro
Julkaisussa Mol Ther (2018)

A novel genomic inversion in Wiskott-Aldrich–associated autoinflammation

Tekijä Brigida, Immacolata, Scaramuzza, Samantha, Lazarevic, Dejan, Cittaro, Davide, Ferrua, Francesca, Leonardelli, Lorena, Alessio, Maria, Forma, Ornella, Lanzani, Chiara, Viarengo, Gianluca, Ciceri, Fabio, Jankovic, Momcilo, Pesce, Fernando, Aiuti, Alessandro, Cicalese, Maria Pia
Julkaisussa J Allergy Clin Immunol (2016)

B-cell reconstitution after lentiviral vector–mediated gene therapy in patients with Wiskott-Aldrich syndrome

Tekijä Castiello, Maria Carmina, Scaramuzza, Samantha, Pala, Francesca, Ferrua, Francesca, Uva, Paolo, Brigida, Immacolata, Sereni, Lucia, van der Burg, Mirjam, Ottaviano, Giorgio, Albert, Michael H., Grazia Roncarolo, Maria, Naldini, Luigi, Aiuti, Alessandro, Villa, Anna, Bosticardo, Marita
Julkaisussa J Allergy Clin Immunol (2015)

Pioglitazone as a novel therapeutic approach in chronic granulomatous disease

Tekijä Migliavacca, Maddalena, Assanelli, Andrea, Ferrua, Francesca, Cicalese, Maria Pia, Biffi, Alessandra, Frittoli, Marta, Silvani, Paolo, Chidini, Giovanna, Calderini, Edoardo, Mandelli, Anna, Camporesi, Anna, Milani, Raffaella, Farinelli, Giada, Nicoletti, Roberto, Ciceri, Fabio, Aiuti, Alessandro, Bernardo, Maria Ester
Julkaisussa J Allergy Clin Immunol (2016)

First Occurrence of Plasmablastic Lymphoma in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Disease Patient and Review of the Literature

Tekijä Migliavacca, Maddalena, Assanelli, Andrea, Ponzoni, Maurilio, Pajno, Roberta, Barzaghi, Federica, Giglio, Fabio, Ferrua, Francesca, Frittoli, Marta, Brigida, Immacolata, Dionisio, Francesca, Nicoletti, Roberto, Casiraghi, Miriam, Roncarolo, Maria Grazia, Doglioni, Claudio, Peccatori, Jacopo, Ciceri, Fabio, Cicalese, Maria Pia, Aiuti, Alessandro
Julkaisussa Front Immunol (2018)

Urogenital Abnormalities in Adenosine Deaminase Deficiency

Tekijä Pajno, Roberta, Pacillo, Lucia, Recupero, Salvatore, Cicalese, Maria P., Ferrua, Francesca, Barzaghi, Federica, Ricci, Silvia, Marzollo, Antonio, Pecorelli, Silvia, Azzari, Chiara, Finocchi, Andrea, Cancrini, Caterina, Di Matteo, Gigliola, Russo, Gianni, Alfano, Massimo, Lesma, Arianna, Salonia, Andrea, Adams, Stuart, Booth, Claire, Aiuti, Alessandro
Julkaisussa J Clin Immunol (2020)

Premature Senescence and Increased Oxidative Stress in the Thymus of Down Syndrome Patients

Tekijä Marcovecchio, Genni Enza, Ferrua, Francesca, Fontana, Elena, Beretta, Stefano, Genua, Marco, Bortolomai, Ileana, Conti, Anastasia, Montin, Davide, Cascarano, Maria Teresa, Bergante, Sonia, D’Oria, Veronica, Giamberti, Alessandro, Amodio, Donato, Cancrini, Caterina, Carotti, Adriano, Di Micco, Raffaella, Merelli, Ivan, Bosticardo, Marita, Villa, Anna
Julkaisussa Front Immunol (2021)

Thymic Epithelium Abnormalities in DiGeorge and Down Syndrome Patients Contribute to Dysregulation in T Cell Development

Tekijä Marcovecchio, Genni Enza, Bortolomai, Ileana, Ferrua, Francesca, Fontana, Elena, Imberti, Luisa, Conforti, Erika, Amodio, Donato, Bergante, Sonia, Macchiarulo, Giulia, D'Oria, Veronica, Conti, Francesca, Di Cesare, Silvia, Fousteri, Georgia, Carotti, Adriano, Giamberti, Alessandro, Poliani, Pietro Luigi, Notarangelo, Luigi D., Cancrini, Caterina, Villa, Anna, Bosticardo, Marita
Julkaisussa Front Immunol (2019)

Gene Modification and Three‐Dimensional Scaffolds as Novel Tools to Allow the Use of Postnatal Thymic Epithelial Cells for Thymus Regeneration Approaches

Tekijä Bortolomai, Ileana, Sandri, Monica, Draghici, Elena, Fontana, Elena, Campodoni, Elisabetta, Marcovecchio, Genni Enza, Ferrua, Francesca, Perani, Laura, Spinelli, Antonello, Canu, Tamara, Catucci, Marco, Di Tomaso, Tiziano, Sergi Sergi, Lucia, Esposito, Antonio, Lombardo, Angelo, Naldini, Luigi, Tampieri, Anna, Hollander, Georg A., Villa, Anna, Bosticardo, Marita
Julkaisussa Stem Cells Transl Med (2019)

In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases

Tekijä Biasco, Luca, Pellin, Danilo, Scala, Serena, Dionisio, Francesca, Basso-Ricci, Luca, Leonardelli, Lorena, Scaramuzza, Samantha, Baricordi, Cristina, Ferrua, Francesca, Cicalese, Maria Pia, Giannelli, Stefania, Neduva, Victor, Dow, David J., Schmidt, Manfred, Von Kalle, Christof, Roncarolo, Maria Grazia, Ciceri, Fabio, Vicard, Paola, Wit, Ernst, Di Serio, Clelia, Naldini, Luigi, Aiuti, Alessandro
Julkaisussa Cell Stem Cell (2016)

Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper‐IgM syndrome

Tekijä Vavassori, Valentina, Mercuri, Elisabetta, Marcovecchio, Genni E, Castiello, Maria C, Schiroli, Giulia, Albano, Luisa, Margulies, Carrie, Buquicchio, Frank, Fontana, Elena, Beretta, Stefano, Merelli, Ivan, Cappelleri, Andrea, Rancoita, Paola MV, Lougaris, Vassilios, Plebani, Alessandro, Kanariou, Maria, Lankester, Arjan, Ferrua, Francesca, Scanziani, Eugenio, Cotta‐Ramusino, Cecilia, Villa, Anna, Naldini, Luigi, Genovese, Pietro
Julkaisussa EMBO Mol Med (2021)

B-cell development and functions and therapeutic options in adenosine deaminase–deficient patients

Tekijä Brigida, Immacolata, Sauer, Aisha V., Ferrua, Francesca, Giannelli, Stefania, Scaramuzza, Samantha, Pistoia, Valentina, Castiello, Maria Carmina, Barendregt, Barbara H., Cicalese, Maria Pia, Casiraghi, Miriam, Brombin, Chiara, Puck, Jennifer, Müller, Klaus, Notarangelo, Lucia Dora, Montin, Davide, van Montfrans, Joris M., Roncarolo, Maria Grazia, Traggiai, Elisabetta, van Dongen, Jacques J. M., van der Burg, Mirjam, Aiuti, Alessandro
Julkaisussa J Allergy Clin Immunol (2014)

Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strim...

Tekijä Tucci, Francesca, Calbi, Valeria, Barzaghi, Federica, Migliavacca, Maddalena, Ferrua, Francesca, Bernardo, Maria Ester, Canarutto, Daniele, Consiglieri, Giulia, Recupero, Salvatore, Calzatini, Francesco, Gabaldo, Michela, Lucano, Caterina, Casiraghi, Miriam, Darin, Silvia, Dionisio, Francesca, Marktel, Sarah, Cestone, Enza, Finazzi, Renato, Mieli‐Vergani, Giorgina, Boeri, Enzo, Appleby, Jonathan, Abd Elaziz, Dalia, Ciceri, Fabio, Aiuti, Alessandro, Cicalese, Maria Pia
Julkaisussa Hepatology (2018)

Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome

Tekijä Sereni, Lucia, Castiello, Maria Carmina, Di Silvestre, Dario, Della Valle, Patrizia, Brombin, Chiara, Ferrua, Francesca, Cicalese, Maria Pia, Pozzi, Loris, Migliavacca, Maddalena, Bernardo, Maria Ester, Pignata, Claudio, Farah, Roula, Notarangelo, Lucia Dora, Marcus, Nufar, Cattaneo, Lorella, Spinelli, Marco, Giannelli, Stefania, Bosticardo, Marita, van Rossem, Koen, D'Angelo, Armando, Aiuti, Alessandro, Mauri, Pierluigi, Villa, Anna
Julkaisussa J Allergy Clin Immunol (2019)

Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis- related graft failure and disseminated bacillus Calmette-Guérin infection

Tekijä Tucci, Francesca, Gallo, Vera, Barzaghi, Federica, Ferrua, Francesca, Migliavacca, Maddalena, Calbi, Valeria, Doglio, Matteo, Fratini, Elena Sophia, Karakas, Zeynep, Guner, Sukru, Zambelli, Matilde, Parisi, Cristina, Milani, Raffaella, Gattillo, Salvatore, Mazzi, Benedetta, Oltolini, Chiara, Barbera, Maurizio, Baldoli, Cristina, Cirillo, Daniela Maria, Asnaghi, Veronica, de Min, Cristina, Cicalese, Maria Pia, Ciceri, Fabio, Aiuti, Alessandro, Bernardo, Maria Ester
Julkaisussa Haematologica (2020)

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Tekijä Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Pajno, Roberta, Barzaghi, Federica, Giannelli, Stefania, Dionisio, Francesca, Brigida, Immacolata, Bonopane, Marco, Casiraghi, Miriam, Tabucchi, Antonella, Carlucci, Filippo, Grunebaum, Eyal, Adeli, Mehdi, Bredius, Robbert G., Puck, Jennifer M., Stepensky, Polina, Tezcan, Ilhan, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Ciceri, Fabio, Roncarolo, Maria Grazia, Aiuti, Alessandro
Julkaisussa Blood (2016)

Alterations in the brain adenosine metabolism cause behavioral and neurological impairment in ADA-deficient mice and patients

Tekijä Sauer, Aisha V., Hernandez, Raisa Jofra, Fumagalli, Francesca, Bianchi, Veronica, Poliani, Pietro L., Dallatomasina, Chiara, Riboni, Elisa, Politi, Letterio S., Tabucchi, Antonella, Carlucci, Filippo, Casiraghi, Miriam, Carriglio, Nicola, Cominelli, Manuela, Forcellini, Carlo Alberto, Barzaghi, Federica, Ferrua, Francesca, Minicucci, Fabio, Medaglini, Stefania, Leocani, Letizia, la Marca, Giancarlo, Notarangelo, Lucia D., Azzari, Chiara, Comi, Giancarlo, Baldoli, Cristina, Canale, Sabrina, Sessa, Maria, D’Adamo, Patrizia, Aiuti, Alessandro
Julkaisussa Sci Rep (2017)