| Hot spots of retroviral integration in human CD34+ hematopoietic cells C Cattoglio, G Facchini, D Sartori, A Antonelli, A Miccio, B Cassani, ... Blood, The Journal of the American Society of Hematology 110 (6), 1770-1778, 2007 | 333 | 2007 |
| Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus C Antoniani, V Meneghini, A Lattanzi, T Felix, O Romano, E Magrin, ... Blood, The Journal of the American Society of Hematology 131 (17), 1960-1973, 2018 | 210 | 2018 |
| Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts A Moiani, Y Paleari, D Sartori, R Mezzadra, A Miccio, C Cattoglio, ... The Journal of clinical investigation 122 (5), 1653-1666, 2012 | 203 | 2012 |
| Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replication F Di Nunzio, T Fricke, A Miccio, JC Valle-Casuso, P Perez, P Souque, ... Virology 440 (1), 8-18, 2013 | 193 | 2013 |
| In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of β-thalassemia A Miccio, R Cesari, F Lotti, C Rossi, F Sanvito, M Ponzoni, SJE Routledge, ... Proceedings of the National Academy of Sciences 105 (30), 10547-10552, 2008 | 182 | 2008 |
| NuRD mediates activating and repressive functions of GATA‐1 and FOG‐1 during blood development A Miccio, Y Wang, W Hong, GD Gregory, H Wang, X Yu, JK Choi, S Shelat, ... The EMBO journal 29 (2), 442-456, 2010 | 179 | 2010 |
| Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges M Cavazzana, FD Bushman, A Miccio, I André-Schmutz, E Six Nature reviews Drug discovery 18 (6), 447-462, 2019 | 160 | 2019 |
| Gene therapy for β-hemoglobinopathies M Cavazzana, C Antoniani, A Miccio Molecular therapy 25 (5), 1142-1154, 2017 | 148 | 2017 |
| Transcription factor binding sites are genetic determinants of retroviral integration in the human genome B Felice, C Cattoglio, D Cittaro, A Testa, A Miccio, G Ferrari, L Luzi, ... PloS one 4 (2), e4571, 2009 | 129 | 2009 |
| Optimization of CRISPR/Cas9 delivery to human hematopoietic stem and progenitor cells for therapeutic genomic rearrangements A Lattanzi, V Meneghini, G Pavani, F Amor, S Ramadier, T Felix, ... Molecular Therapy 27 (1), 137-150, 2019 | 122 | 2019 |
| Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype L Weber, G Frati, T Felix, G Hardouin, A Casini, C Wollenschlaeger, ... Science advances 6 (7), eaay9392, 2020 | 117 | 2020 |
| Hypomorphic mutation of the TALE gene Prep1 (pKnox1) causes a major reduction of Pbx and Meis proteins and a pleiotropic embryonic phenotype E Ferretti, JC Villaescusa, P Di Rosa, LC Fernandez-Diaz, E Longobardi, ... Molecular and cellular biology, 2006 | 116 | 2006 |
| Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion C Lagresle-Peyrou, F Lefrère, E Magrin, JA Ribeil, O Romano, L Weber, ... haematologica 103 (5), 778, 2018 | 111 | 2018 |
| Phosphorylation of synapsin I by cAMP-dependent protein kinase controls synaptic vesicle dynamics in developing neurons D Bonanomi, A Menegon, A Miccio, G Ferrari, A Corradi, HT Kao, ... Journal of Neuroscience 25 (32), 7299-7308, 2005 | 101 | 2005 |
| Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies E Magrin, A Miccio, M Cavazzana Blood, The Journal of the American Society of Hematology 134 (15), 1203-1213, 2019 | 99 | 2019 |
| Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial E Magrin, M Semeraro, N Hebert, L Joseph, A Magnani, A Chalumeau, ... Nature Medicine 28 (1), 81-88, 2022 | 71 | 2022 |
| Gene therapy of hemoglobinopathies: progress and future challenges Y Ikawa, A Miccio, E Magrin, JL Kwiatkowski, S Rivella, M Cavazzana Human Molecular Genetics 28 (R1), R24-R30, 2019 | 70 | 2019 |
| FOG1 requires NuRD to promote hematopoiesis and maintain lineage fidelity within the megakaryocytic-erythroid compartment GD Gregory, A Miccio, A Bersenev, Y Wang, W Hong, Z Zhang, M Poncz, ... Blood, The Journal of the American Society of Hematology 115 (11), 2156-2166, 2010 | 70 | 2010 |
| Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells G Pavani, A Fabiano, M Laurent, F Amor, E Cantelli, A Chalumeau, ... Blood advances 5 (5), 1137-1153, 2021 | 59 | 2021 |
| Dynamic transcriptional and epigenetic regulation of human epidermal keratinocyte differentiation A Cavazza, A Miccio, O Romano, L Petiti, GM Tagliazucchi, C Peano, ... Stem cell reports 6 (4), 618-632, 2016 | 58 | 2016 |
