CASTIELLO, MARIA CARMINA
CASTIELLO, MARIA CARMINA
Istituto di Ricerca Genetica e Biomedica - IRGB - Sede Secondaria Milano
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency
2024 Castiello, Maria Carmina; Brandas, Chiara; Ferrari, Samuele; Porcellini, Simona; Sacchetti, Nicolò; Canarutto, Daniele; Draghici, Elena; Merelli, Ivan; Barcella, Matteo; Pelosi, Gabriele; Vavassori, Valentina; Varesi, Angelica; Jacob, Aurelien; Scala, Serena; Basso Ricci, Luca; Paulis, Marianna; Strina, Dario; Di Verniere, Martina; Sergi Sergi, Lucia; Serafini, Marta; Holland, Steven M.; Bergerson, Jenna R. E.; De Ravin, Suk See; Malech, Harry L.; Pala, Francesca; Bosticardo, Marita; Brombin, Chiara; Cugnata, Federica; Calzoni, Enrica; Crooks, Gay M.; Notarangelo, Luigi D.; Genovese, Pietro; Naldini, Luigi; Villa, Anna
HyperIgE in hypomorphic recombination-activating gene defects
2023 Castiello, MARIA CARMINA; Brandas, Chiara; Capo, Valentina; Villa, Anna
Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations
2023 Castiello, Maria Carmina; Di Verniere, Martina; Draghici, Elena; Fontana, Elena; Penna, Sara; Sereni, Lucia; Zecchillo, Alessandra; Minuta, Denise; Uva, Paolo; Zahn, Marco; GilFarina, Irene; Annoni, Andrea; Iaia, Silvia; Ott de Bruin, Lisa M.; Notarangelo, Luigi D.; PikeOverzet, Karin; Staal, Frank J.T.; Villa, Anna; Capo, Valentina
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
2022 Ferrari, Samuele; Jacob, Aurelien; Cesana, Daniela; Laugel, Marianne; Beretta, Stefano; Varesi, Angelica; Unali, Giulia; Conti, Anastasia; Canarutto, Daniele; Albano, Luisa; Calabria, Andrea; Vavassori, Valentina; Cipriani, Carlo; Castiello, Maria Carmina; Esposito, Simona; Brombin, Chiara; Cugnata, Federica; Adjali, Oumeya; Ayuso, Eduard; Merelli, Ivan; Villa, Anna; Di Micco, Raffaella; KajasteRudnitski, Anna; Montini, Eugenio; PenaudBudloo, Magalie; Naldini, Luigi
Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation
2021 Ferrari, S.; Vavassori, V.; Canarutto, D.; Jacob, A.; Castiello, M. C.; Javed, A. O.; Genovese, P.
Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome
2021 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 M.V.; Lougaris, Vassilios; Plebani, Alessandro; Kanariou, Maria; Lankester, Arjan; Ferrua, Francesca; Scanziani, Eugenio; CottaRamusino, Cecilia; Villa, Anna; Naldini, Luigi; Genovese, Pietro
Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency
2020 Castiello M.C.; Bosticardo M.; Sacchetti N.; Calzoni E.; Fontana E.; Yamazaki Y.; Draghici E.; Corsino C.; Bortolomai I.; Sereni L.; Yu H.H.; Uva P.; Palchaudhuri R.; Scadden D.T.; Villa A.; Notarangelo L.D.
Innovative Cell-Based Therapies and Conditioning to Cure RAG Deficiency
2020 Villa, A; Capo, V; Castiello, Mc
Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation
2018 Capo V.; Castiello M.C.; Fontana E.; Penna S.; Bosticardo M.; Draghici E.; Poliani L.P.; Sergi Sergi L.; Rigoni R.; Cassani B.; Zanussi M.; Carrera P.; Uva P.; Dobbs K.; Sacchetti N.; Notarangelo L.D.; van Til N.P.; Wagemaker G.; Villa A.
In vivo chronic stimulation unveils autoreactive potential of Wiskott-Aldrich syndrome protein-deficient b cells
2017 Castiello M.C.; Pala F.; Sereni L.; Draghici E.; Inverso D.; Sauer A.V.; Schena F.; Fontana E.; Radaelli E.; Uva P.; CervantesLuevano K.E.; Benvenuti F.; Poliani P.L.; Iannacone M.; Traggiai E.; Villa A.; Bosticardo M.
Platelets in Wiskott-Aldrich syndrome: victims or executioners?
2017 Sereni, Lucia; Castiello, Maria Carmina; Villa, Anna
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
2017 Schiroli, G; Ferrari, S; Conway, A; Jacob, A; Capo, V; Albano, L; Plati, T; Castiello, Mc; Sanvito, F; Gennery, Ar; Bovolenta, C; Palchaudhuri, R; Scadden, Dt; Holmes, Mc; Villa, A; Sitia, G; Lombardo, A; Genovese, P; Naldini, L
Intestinal microbiota sustains inflammation and autoimmunity induced by hypomorphic RAG defects
2016 Rigoni, R; Fontana, E; Guglielmetti, S; Fosso, B; D'Erchia, Am; Maina, V; Taverniti, V; Castiello, Mc; Mantero, S; Pacchiana, G; Musio, S; Pedotti, R; Selmi, C; Rodrigo Mora, J; Pesole, G; Vezzoni, P; Poliani, Pl; Grassi, F; Villa, A; Cassani, B
B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome
2015 Castiello, Mc; Scaramuzza, S; Pala, F; Ferrua, F; Uva, P; Brigida, I; Sereni, L; Van Der Burg, M; Ottaviano, G; Albert, Mh; Grazia Roncarolo, M; Naldini, L; Aiuti, A; Villa, A; Bosticardo, M
IL-10 Critically Modulates B Cell Responsiveness in Rankl(-/-) Mice
2015 Marrella, VERONICA AGATA; Lo Iacono, Nadia; Fontana, Elena; Sobacchi, Cristina; Sic, Heiko; Schena, Francesca; Sereni, Lucia; Castiello Maria, Carmina; Poliani Pietro, Luigi; Vezzoni, PAOLO MARIA; Cassani, Barbara; Traggiai, Elisabetta; Villa, Anna
B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients
2014 Brigida, I.; Sauer, A. V.; Ferrua, F.; Giannelli, S.; Scaramuzza, S.; Pistoia, V.; Castiello, M. C.; Barendregt, B. H.; Cicalese, M. P.; Casiraghi, M.; Brombin, C.; Puck, J.; Muller, K.; Notarangelo, L. D.; Montin, D.; Van Montfrans, J. M.; Roncarolo, M. G.; Traggiai, E.; Van Dongen, J. J. M.; Van Der Burg, M.; Aiuti, A.
Wiskott-Aldrich syndrome protein deficiency in natural killer and dendritic cells affects antitumor immunity
2014 Catucci, M; Zanoni, I; Draghici, E; Bosticardo, M; Castiello, Mc; Venturini, M; Cesana, D; Montini, E; Ponzoni, M; Granucci, F; Villa, A
Wiskott-Aldrich Syndrome protein deficiency perturbs the homeostasis of B-cell compartment in humans
2014 Castiello M.C.; Bosticardo M.; Pala F.; Catucci M.; Chamberlain N.; Van Zelm M.C.; Driessen G.J.; Pac M.; Bernatowska E.; Scaramuzza S.; Aiuti A.; Sauer A.V.; Traggiai E.; Meffre E.; Villa A.; Van der Burg M.
SOCS1 gene transfer accelerates the transition to heart failure through the inhibition of the gp130/JAK/STAT pathway
2012 Cittadini, A.; Monti, M. G.; Iaccarino, G.; Castiello, M. C.; Baldi, A.; Bossone, E.; Longobardi, S.; Marra, A. M.; Petrillo, V.; Saldamarco, L.; During, M. J.; Sacca, L.; Condorelli, G.
Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: Analysis of function and distribution in lymphoid organs.
2010 Trifari, S; Scaramuzza, S; Catucci, M; Ponzoni, M; Mollica, L; Chiesa, R; Cattaneo, F; Lafouresse, F; Calvez, R; Vermi, W; Medicina, D; Castiello, Mc; Marangoni, F; Bosticardo, M; Doglioni, C; Caniglia, M; Aiuti, A; Villa, A; Roncarolo, Mg; Dupré, L
| Titolo | Data di pubblicazione | Autore(i) | File |
|---|---|---|---|
| Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency | 1-gen-2024 | Castiello, Maria Carmina; Brandas, Chiara; Ferrari, Samuele; Porcellini, Simona; Sacchetti, Nicolò; Canarutto, Daniele; Draghici, Elena; Merelli, Ivan; Barcella, Matteo; Pelosi, Gabriele; Vavassori, Valentina; Varesi, Angelica; Jacob, Aurelien; Scala, Serena; Basso Ricci, Luca; Paulis, Marianna; Strina, Dario; Di Verniere, Martina; Sergi Sergi, Lucia; Serafini, Marta; Holland, Steven M.; Bergerson, Jenna R. E.; De Ravin, Suk See; Malech, Harry L.; Pala, Francesca; Bosticardo, Marita; Brombin, Chiara; Cugnata, Federica; Calzoni, Enrica; Crooks, Gay M.; Notarangelo, Luigi D.; Genovese, Pietro; Naldini, Luigi; Villa, Anna | |
| HyperIgE in hypomorphic recombination-activating gene defects | 1-gen-2023 | Castiello, MARIA CARMINA; Brandas, Chiara; Capo, Valentina; Villa, Anna | |
| Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations | 1-gen-2023 | Castiello, Maria Carmina; Di Verniere, Martina; Draghici, Elena; Fontana, Elena; Penna, Sara; Sereni, Lucia; Zecchillo, Alessandra; Minuta, Denise; Uva, Paolo; Zahn, Marco; GilFarina, Irene; Annoni, Andrea; Iaia, Silvia; Ott de Bruin, Lisa M.; Notarangelo, Luigi D.; PikeOverzet, Karin; Staal, Frank J.T.; Villa, Anna; Capo, Valentina | |
| Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells | 1-gen-2022 | Ferrari, Samuele; Jacob, Aurelien; Cesana, Daniela; Laugel, Marianne; Beretta, Stefano; Varesi, Angelica; Unali, Giulia; Conti, Anastasia; Canarutto, Daniele; Albano, Luisa; Calabria, Andrea; Vavassori, Valentina; Cipriani, Carlo; Castiello, Maria Carmina; Esposito, Simona; Brombin, Chiara; Cugnata, Federica; Adjali, Oumeya; Ayuso, Eduard; Merelli, Ivan; Villa, Anna; Di Micco, Raffaella; KajasteRudnitski, Anna; Montini, Eugenio; PenaudBudloo, Magalie; Naldini, Luigi | |
| Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation | 1-gen-2021 | Ferrari, S.; Vavassori, V.; Canarutto, D.; Jacob, A.; Castiello, M. C.; Javed, A. O.; Genovese, P. | |
| Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome | 1-gen-2021 | 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 M.V.; Lougaris, Vassilios; Plebani, Alessandro; Kanariou, Maria; Lankester, Arjan; Ferrua, Francesca; Scanziani, Eugenio; CottaRamusino, Cecilia; Villa, Anna; Naldini, Luigi; Genovese, Pietro | |
| Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency | 1-gen-2020 | Castiello M.C.; Bosticardo M.; Sacchetti N.; Calzoni E.; Fontana E.; Yamazaki Y.; Draghici E.; Corsino C.; Bortolomai I.; Sereni L.; Yu H.H.; Uva P.; Palchaudhuri R.; Scadden D.T.; Villa A.; Notarangelo L.D. | |
| Innovative Cell-Based Therapies and Conditioning to Cure RAG Deficiency | 1-gen-2020 | Villa, A; Capo, V; Castiello, Mc | |
| Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation | 1-gen-2018 | Capo V.; Castiello M.C.; Fontana E.; Penna S.; Bosticardo M.; Draghici E.; Poliani L.P.; Sergi Sergi L.; Rigoni R.; Cassani B.; Zanussi M.; Carrera P.; Uva P.; Dobbs K.; Sacchetti N.; Notarangelo L.D.; van Til N.P.; Wagemaker G.; Villa A. | |
| In vivo chronic stimulation unveils autoreactive potential of Wiskott-Aldrich syndrome protein-deficient b cells | 1-gen-2017 | Castiello M.C.; Pala F.; Sereni L.; Draghici E.; Inverso D.; Sauer A.V.; Schena F.; Fontana E.; Radaelli E.; Uva P.; CervantesLuevano K.E.; Benvenuti F.; Poliani P.L.; Iannacone M.; Traggiai E.; Villa A.; Bosticardo M. | |
| Platelets in Wiskott-Aldrich syndrome: victims or executioners? | 1-gen-2017 | Sereni, Lucia; Castiello, Maria Carmina; Villa, Anna | |
| Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1 | 1-gen-2017 | Schiroli, G; Ferrari, S; Conway, A; Jacob, A; Capo, V; Albano, L; Plati, T; Castiello, Mc; Sanvito, F; Gennery, Ar; Bovolenta, C; Palchaudhuri, R; Scadden, Dt; Holmes, Mc; Villa, A; Sitia, G; Lombardo, A; Genovese, P; Naldini, L | |
| Intestinal microbiota sustains inflammation and autoimmunity induced by hypomorphic RAG defects | 1-gen-2016 | Rigoni, R; Fontana, E; Guglielmetti, S; Fosso, B; D'Erchia, Am; Maina, V; Taverniti, V; Castiello, Mc; Mantero, S; Pacchiana, G; Musio, S; Pedotti, R; Selmi, C; Rodrigo Mora, J; Pesole, G; Vezzoni, P; Poliani, Pl; Grassi, F; Villa, A; Cassani, B | |
| B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome | 1-gen-2015 | Castiello, Mc; Scaramuzza, S; Pala, F; Ferrua, F; Uva, P; Brigida, I; Sereni, L; Van Der Burg, M; Ottaviano, G; Albert, Mh; Grazia Roncarolo, M; Naldini, L; Aiuti, A; Villa, A; Bosticardo, M | |
| IL-10 Critically Modulates B Cell Responsiveness in Rankl(-/-) Mice | 1-gen-2015 | Marrella, VERONICA AGATA; Lo Iacono, Nadia; Fontana, Elena; Sobacchi, Cristina; Sic, Heiko; Schena, Francesca; Sereni, Lucia; Castiello Maria, Carmina; Poliani Pietro, Luigi; Vezzoni, PAOLO MARIA; Cassani, Barbara; Traggiai, Elisabetta; Villa, Anna | |
| B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients | 1-gen-2014 | Brigida, I.; Sauer, A. V.; Ferrua, F.; Giannelli, S.; Scaramuzza, S.; Pistoia, V.; Castiello, M. C.; Barendregt, B. H.; Cicalese, M. P.; Casiraghi, M.; Brombin, C.; Puck, J.; Muller, K.; Notarangelo, L. D.; Montin, D.; Van Montfrans, J. M.; Roncarolo, M. G.; Traggiai, E.; Van Dongen, J. J. M.; Van Der Burg, M.; Aiuti, A. | |
| Wiskott-Aldrich syndrome protein deficiency in natural killer and dendritic cells affects antitumor immunity | 1-gen-2014 | Catucci, M; Zanoni, I; Draghici, E; Bosticardo, M; Castiello, Mc; Venturini, M; Cesana, D; Montini, E; Ponzoni, M; Granucci, F; Villa, A | |
| Wiskott-Aldrich Syndrome protein deficiency perturbs the homeostasis of B-cell compartment in humans | 1-gen-2014 | Castiello M.C.; Bosticardo M.; Pala F.; Catucci M.; Chamberlain N.; Van Zelm M.C.; Driessen G.J.; Pac M.; Bernatowska E.; Scaramuzza S.; Aiuti A.; Sauer A.V.; Traggiai E.; Meffre E.; Villa A.; Van der Burg M. | |
| SOCS1 gene transfer accelerates the transition to heart failure through the inhibition of the gp130/JAK/STAT pathway | 1-gen-2012 | Cittadini, A.; Monti, M. G.; Iaccarino, G.; Castiello, M. C.; Baldi, A.; Bossone, E.; Longobardi, S.; Marra, A. M.; Petrillo, V.; Saldamarco, L.; During, M. J.; Sacca, L.; Condorelli, G. | |
| Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: Analysis of function and distribution in lymphoid organs. | 1-gen-2010 | Trifari, S; Scaramuzza, S; Catucci, M; Ponzoni, M; Mollica, L; Chiesa, R; Cattaneo, F; Lafouresse, F; Calvez, R; Vermi, W; Medicina, D; Castiello, Mc; Marangoni, F; Bosticardo, M; Doglioni, C; Caniglia, M; Aiuti, A; Villa, A; Roncarolo, Mg; Dupré, L |