| Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1 G Schiroli, S Ferrari, A Conway, A Jacob, V Capo, L Albano, T Plati, ... Science translational medicine 9 (411), eaan0820, 2017 | 218 | 2017 |
| Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis M Chiriaco, G Farinelli, V Capo, E Zonari, S Scaramuzza, G Di Matteo, ... Molecular Therapy 22 (8), 1472-1483, 2014 | 82 | 2014 |
| One disease, many genes: implications for the treatment of osteopetroses S Penna, V Capo, E Palagano, C Sobacchi, A Villa Frontiers in Endocrinology 10, 85, 2019 | 73 | 2019 |
| Lentiviral vectors for the treatment of primary immunodeficiencies G Farinelli, V Capo, S Scaramuzza, A Aiuti Journal of inherited metabolic disease 37, 525-533, 2014 | 39 | 2014 |
| Autosomal recessive osteopetrosis: mechanisms and treatments S Penna, A Villa, V Capo Disease Models & Mechanisms 14 (5), dmm048940, 2021 | 37 | 2021 |
| Cysteine and hydrophobic residues in CDR3 serve as distinct T-cell self-reactivity indices SR Daley, HF Koay, K Dobbs, M Bosticardo, RC Wirasinha, F Pala, ... Journal of Allergy and Clinical Immunology 144 (1), 333-336, 2019 | 36 | 2019 |
| Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation V Capo, MC Castiello, E Fontana, S Penna, M Bosticardo, E Draghici, ... Journal of Allergy and Clinical Immunology 142 (3), 928-941. e8, 2018 | 33 | 2018 |
| Murine Rankl−/− Mesenchymal Stromal Cells Display an Osteogenic Differentiation Defect Improved by a RANKL-Expressing Lentiviral Vector F Schena, C Menale, E Caci, L Diomede, E Palagano, C Recordati, ... Stem Cells 35 (5), 1365-1377, 2017 | 29 | 2017 |
| Expanded circulating hematopoietic stem/progenitor cells as novel cell source for the treatment of TCIRG1 osteopetrosis V Capo, S Penna, I Merelli, M Barcella, S Scala, L Basso-Ricci, E Draghici, ... Haematologica 106 (1), 74, 2021 | 25 | 2021 |
| Innovative cell-based therapies and conditioning to cure RAG deficiency A Villa, V Capo, MC Castiello Frontiers in Immunology 11, 607926, 2020 | 16 | 2020 |
| Osteoclast rich osteopetrosis due to defects in the TCIRG1 gene V Capo, M Abinun, A Villa Bone 165, 116519, 2022 | 12 | 2022 |
| Serratia marcescens osteomyelitis in a newborn with chronic granulomatous disease I Salfa, N Cantarutti, G Angelino, G Di Matteo, V Capo, G Farinelli, ... The Pediatric infectious disease journal 32 (8), 926, 2013 | 12 | 2013 |
| Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1. Sci Transl Med 2017; 9 G Schiroli, S Ferrari, A Conway, A Jacob, V Capo, L Albano | 6 | |
| 481. Targeted genome editing in Mouse hematopoietic stem/progenitor cells (HSPC) to model gene correction of SCID-X1 G Schiroli, P Genovese, MC Castiello, V Capo, PD Gregory, MC Holmes, ... Molecular Therapy 23, S191, 2015 | 4 | 2015 |
| Skeletal damage and cross-correction in MPSIH HSPC-gene therapy L Santi, S Crippa, V Capo, S Penna, G De Ponti, M Berti, M Mancino, ... HUMAN GENE THERAPY 33 (23-24), A3-A4, 2022 | 2 | 2022 |
| Castiello Maria C S Giulia, F Samuele, C Anthony, J Aurelien, C Valentina, A Luisa, ... | 2 | 2017 |
| HyperIgE in hypomorphic recombination-activating gene defects MC Castiello, C Brandas, V Capo, A Villa Current Opinion in Immunology 80, 102279, 2023 | 1 | 2023 |
| Investigation of the bone damage in mucopolysaccharidosis type I Hurler syndrome: pathophysiological mechanisms and the impact of ex vivo gene therapy S Penna, S Crippa, V Capo, L Santi, R Bosotti, M Riminucci, A Corsi, ... Bone Reports 13, 100597, 2020 | 1 | 2020 |
| Modeling, Optimization and Comparative Efficacy of HSC-and T-cell Based Editing Strategies for Treating Hyper IgM Syndrome V Vavassori, E Mercuri, G Marcovecchio, MC Castiello, G Schiroli, ... MOLECULAR THERAPY 28 (4), 407-407, 2020 | 1 | 2020 |
| 42. Correction of SCID-X1 by targeted genome editing of hematopoietic stem/progenitor cells (HSPC) in the mouse model G Schiroli, P Genovese, V Capo, MC Castiello, L Albano, MC Holmes, ... Molecular Therapy 24, S18-S19, 2016 | 1 | 2016 |
