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, M. C.; Brandas, C.; Ferrari, S.; Porcellini, S.; Sacchetti, N.; Canarutto, D.; Draghici, E.; Merelli, I.; Barcella, M.; Pelosi, G.; Vavassori, V.; Varesi, A.; Jacob, A.; Scala, S.; Ricci, L. B.; Paulis, M.; Strina, D.; Di Verniere, M.; Sergi, L. S.; Serafini, M.; Holland, S. M.; Bergerson, J. R. E.; De Ravin, S. S.; Malech, H. L.; Pala, F.; Bosticardo, M.; Brombin, C.; Cugnata, F.; Calzoni, E.; Crooks, G. M.; Notarangelo, L. D.; Genovese, P.; Naldini, L.; Villa, A.
Correcting inborn errors of immunity: From viral mediated gene addition to gene editing
2023 Castiello, M. C.; Ferrari, S.; Villa, A.
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
Thymic Epithelial Cell Alterations and Defective Thymopoiesis Lead to Central and Peripheral Tolerance Perturbation in MHCII Deficiency
2021 Ferrua, F.; Bortolomai, I.; Fontana, E.; Di Silvestre, D.; Rigoni, R.; Marcovecchio, G. E.; Draghici, E.; Brambilla, F.; Castiello, M. C.; Delfanti, G.; Moshous, D.; Picard, C.; Taghon, T.; Bordon, V.; Schulz, A. S.; Schuetz, C.; Giliani, S.; Soresina, A.; Gennery, A. R.; Signa, S.; Davila Saldana, B. J.; Delmonte, O. M.; Notarangelo, L. D.; Roifman, C. M.; Poliani, P. L.; Uva, P.; Mauri, P. L.; Villa, A.; Bosticardo, M.
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
Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome
2019 Sereni L; Castiello MC; Di Silvestre D; Della Valle P; Brombin C; Ferrua F; Cicalese MP; Pozzi L; Migliavacca M; Bernardo ME; Pignata C; Farah R; Notarangelo LD; Marcus N; Cattaneo L; Spinelli M; Giannelli S; Bosticardo M; van Rossem K; D'Angelo A; Aiuti A; Mauri P; Villa A.
Autonomous role of Wiskott-Aldrich Syndrome platelet deficiency in inducing autoimmunity and inflammation.
2018 Sereni L; Castiello MC; Marangoni F; Anselmo A; di Silvestre D; Motta S; Draghici E; Mantero S; Thrasher AJ; Giliani S; Aiuti A; Mauri P; Notarangelo LD; Bosticardo M; Villa A.
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.
Neutrophils drive type I interferon production and autoantibodies in patients with Wiskott-Aldrich syndrome
2018 Cervantes-Luevano, K. E.; Caronni, N.; Castiello, M. C.; Fontana, E.; Piperno, G. M.; Naseem, A.; Uva, P.; Bosticardo, M.; Marcovecchio, G. E.; Notarangelo, L. D.; Cicalese, M. P.; Aiuti, A.; Villa, A.; Benvenuti, F.
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
| 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, M. C.; Brandas, C.; Ferrari, S.; Porcellini, S.; Sacchetti, N.; Canarutto, D.; Draghici, E.; Merelli, I.; Barcella, M.; Pelosi, G.; Vavassori, V.; Varesi, A.; Jacob, A.; Scala, S.; Ricci, L. B.; Paulis, M.; Strina, D.; Di Verniere, M.; Sergi, L. S.; Serafini, M.; Holland, S. M.; Bergerson, J. R. E.; De Ravin, S. S.; Malech, H. L.; Pala, F.; Bosticardo, M.; Brombin, C.; Cugnata, F.; Calzoni, E.; Crooks, G. M.; Notarangelo, L. D.; Genovese, P.; Naldini, L.; Villa, A. | |
| Correcting inborn errors of immunity: From viral mediated gene addition to gene editing | 1-gen-2023 | Castiello, M. C.; Ferrari, S.; Villa, A. | |
| 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 | |
| Thymic Epithelial Cell Alterations and Defective Thymopoiesis Lead to Central and Peripheral Tolerance Perturbation in MHCII Deficiency | 1-gen-2021 | Ferrua, F.; Bortolomai, I.; Fontana, E.; Di Silvestre, D.; Rigoni, R.; Marcovecchio, G. E.; Draghici, E.; Brambilla, F.; Castiello, M. C.; Delfanti, G.; Moshous, D.; Picard, C.; Taghon, T.; Bordon, V.; Schulz, A. S.; Schuetz, C.; Giliani, S.; Soresina, A.; Gennery, A. R.; Signa, S.; Davila Saldana, B. J.; Delmonte, O. M.; Notarangelo, L. D.; Roifman, C. M.; Poliani, P. L.; Uva, P.; Mauri, P. L.; Villa, A.; Bosticardo, M. | |
| 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 | |
| Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome | 1-gen-2019 | Sereni L; Castiello MC; Di Silvestre D; Della Valle P; Brombin C; Ferrua F; Cicalese MP; Pozzi L; Migliavacca M; Bernardo ME; Pignata C; Farah R; Notarangelo LD; Marcus N; Cattaneo L; Spinelli M; Giannelli S; Bosticardo M; van Rossem K; D'Angelo A; Aiuti A; Mauri P; Villa A. | |
| Autonomous role of Wiskott-Aldrich Syndrome platelet deficiency in inducing autoimmunity and inflammation. | 1-gen-2018 | Sereni L; Castiello MC; Marangoni F; Anselmo A; di Silvestre D; Motta S; Draghici E; Mantero S; Thrasher AJ; Giliani S; Aiuti A; Mauri P; Notarangelo LD; Bosticardo M; Villa A. | |
| 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. | |
| Neutrophils drive type I interferon production and autoantibodies in patients with Wiskott-Aldrich syndrome | 1-gen-2018 | Cervantes-Luevano, K. E.; Caronni, N.; Castiello, M. C.; Fontana, E.; Piperno, G. M.; Naseem, A.; Uva, P.; Bosticardo, M.; Marcovecchio, G. E.; Notarangelo, L. D.; Cicalese, M. P.; Aiuti, A.; Villa, A.; Benvenuti, F. | |
| 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 |