Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery A Lombardo, P Genovese, CM Beausejour, S Colleoni, YL Lee, KA Kim, ... Nature biotechnology 25 (11), 1298-1306, 2007 | 1163 | 2007 |
An unbiased genome-wide analysis of zinc-finger nuclease specificity R Gabriel, A Lombardo, A Arens, JC Miller, P Genovese, C Kaeppel, ... Nature biotechnology 29 (9), 816-823, 2011 | 729 | 2011 |
Targeted genome editing in human repopulating haematopoietic stem cells P Genovese, G Schiroli, G Escobar, T Di Tomaso, C Firrito, A Calabria, ... Nature 510 (7504), 235-240, 2014 | 699 | 2014 |
Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer E Provasi, P Genovese, A Lombardo, Z Magnani, PQ Liu, A Reik, V Chu, ... Nature medicine 18 (5), 807-815, 2012 | 566 | 2012 |
Inheritable silencing of endogenous genes by hit-and-run targeted epigenetic editing A Amabile, A Migliara, P Capasso, M Biffi, D Cittaro, L Naldini, ... Cell 167 (1), 219-232. e14, 2016 | 466 | 2016 |
Site-specific integration and tailoring of cassette design for sustainable gene transfer A Lombardo, D Cesana, P Genovese, B Di Stefano, E Provasi, ... Nature methods 8 (10), 861-869, 2011 | 390 | 2011 |
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice BD Brown, A Cantore, A Annoni, LS Sergi, A Lombardo, P Della Valle, ... Blood, The Journal of the American Society of Hematology 110 (13), 4144-4152, 2007 | 340 | 2007 |
Efficient Gene Delivery and Targeted Expression to Hepatocytes In Vivo by Improved Lentiviral Vectors A Follenzi, G Sabatino, A Lombardo, C Boccaccio, L Naldini Human gene therapy 13 (2), 243-260, 2002 | 316 | 2002 |
Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice A Follenzi, M Battaglia, A Lombardo, A Annoni, MG Roncarolo, L Naldini Blood 103 (10), 3700-3709, 2004 | 285 | 2004 |
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 | 216 | 2017 |
The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related … S Camnasio, AD Carri, A Lombardo, I Grad, C Mariotti, A Castucci, ... Neurobiology of disease 46 (1), 41-51, 2012 | 200 | 2012 |
Genomic instability in induced stem cells CE Pasi, A Dereli-Öz, S Negrini, M Friedli, G Fragola, A Lombardo, ... Cell Death & Differentiation 18 (5), 745-753, 2011 | 188 | 2011 |
Contemporary management of acute coronary syndromes: does the practice match the evidence? The global registry of acute coronary events (GRACE) KF Carruthers, OH Dabbous, MD Flather, I Starkey, A Jacob, D Macleod, ... Heart 91 (3), 290-298, 2005 | 152 | 2005 |
TRPML1 links lysosomal calcium to autophagosome biogenesis through the activation of the CaMKKβ/VPS34 pathway A Scotto Rosato, S Montefusco, C Soldati, S Di Paola, A Capuozzo, ... Nature communications 10 (1), 5630, 2019 | 139 | 2019 |
iPSC-derived neural precursors exert a neuroprotective role in immune-mediated demyelination via the secretion of LIF C Laterza, A Merlini, D De Feo, F Ruffini, R Menon, M Onorati, E Fredrickx, ... Nature communications 4 (1), 2597, 2013 | 129 | 2013 |
Loss of transcriptional control over endogenous retroelements during reprogramming to pluripotency M Friedli, P Turelli, A Kapopoulou, B Rauwel, N Castro-Díaz, HM Rowe, ... Genome research 24 (8), 1251-1259, 2014 | 118 | 2014 |
Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice W Piacibello, S Bruno, F Sanavio, S Droetto, M Gunetti, L Ailles, ... Blood, The Journal of the American Society of Hematology 100 (13), 4391-4400, 2002 | 117 | 2002 |
T-cell defects in patients with ARPC1B germline mutations account for combined immunodeficiency I Brigida, M Zoccolillo, MP Cicalese, L Pfajfer, F Barzaghi, S Scala, ... Blood, The Journal of the American Society of Hematology 132 (22), 2362-2374, 2018 | 113 | 2018 |
Methods and compositions for targeted integration MC Holmes, S Yao, L Naldini, AL Lombardo US Patent 8,936,936, 2015 | 106 | 2015 |
Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector CD Domenico, GRD Villani, DD Napoli, EGY Reyero, A Lombardo, ... Human gene therapy 16 (1), 81-90, 2005 | 104 | 2005 |