Cornelia de Lange syndrome mutations in SMC1A or SMC3 affect binding to DNA E Revenkova, ML Focarelli, L Susani, M Paulis, MT Bassi, L Mannini, ... Human molecular genetics 18 (3), 418-427, 2009 | 125 | 2009 |
The K219T-Lamin mutation induces conduction defects through epigenetic inhibition of SCN5A in human cardiac laminopathy N Salvarani, S Crasto, M Miragoli, A Bertero, M Paulis, P Kunderfranco, ... Nature communications 10 (1), 2267, 2019 | 92 | 2019 |
Generation of human induced pluripotent stem cell-derived bona fide neural stem cells for ex vivo gene therapy of metachromatic leukodystrophy V Meneghini, G Frati, D Sala, S De Cicco, M Luciani, C Cavazzin, ... Stem Cells Translational Medicine 6 (2), 352-368, 2017 | 73 | 2017 |
Osteopetrosis rescue upon RANKL administration to Rankl−/− mice: A new therapy for human RANKL‐dependent ARO N Lo Iacono, HC Blair, PL Poliani, V Marrella, F Ficara, B Cassani, ... Journal of Bone and Mineral Research 27 (12), 2501-2510, 2012 | 66 | 2012 |
Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons PG Mazzara, S Muggeo, M Luoni, L Massimino, M Zaghi, PTT Valverde, ... Nature Communications 11 (1), 4178, 2020 | 52 | 2020 |
Cloning of the bovine prion-like Shadoo (SPRN) gene by comparative analysis of the predicted genomic locus C Uboldi, M Paulis, E Guidi, A Bertoni, GPD Meo, A Perucatti, L Iannuzzi, ... Mammalian genome 17, 1130-1139, 2006 | 39 | 2006 |
Claspin inhibition leads to fragile site expression ML Focarelli, S Soza, L Mannini, M Paulis, A Montecucco, A Musio Genes, Chromosomes and Cancer 48 (12), 1083-1090, 2009 | 30 | 2009 |
A pre-screening FISH-based method to detect CRISPR/Cas9 off-targets in mouse embryonic stem cells M Paulis, A Castelli, M Lizier, L Susani, F Lucchini, A Villa, P Vezzoni Scientific reports 5 (1), 12327, 2015 | 27 | 2015 |
Anti-CD3ε mAb improves thymic architecture and prevents autoimmune manifestations in a mouse model of Omenn syndrome: therapeutic implications V Marrella, PL Poliani, E Fontana, A Casati, V Maina, B Cassani, F Ficara, ... Blood, The Journal of the American Society of Hematology 120 (5), 1005-1014, 2012 | 26 | 2012 |
Targeted gene correction in osteopetrotic-induced pluripotent stem cells for the generation of functional osteoclasts T Neri, S Muggeo, M Paulis, ME Caldana, L Crisafulli, D Strina, ... Stem Cell Reports 5 (4), 558-568, 2015 | 25 | 2015 |
Transfer of a human chromosomal vector from a hamster cell line to a mouse embryonic stem cell line M Paulis, M Bensi, D Orioli, C Mondello, G Mazzini, M D'Incalci, C Falcioni, ... Stem Cells 25 (10), 2543-2550, 2007 | 25 | 2007 |
Prion-like Doppel gene (PRND) in the goat: genomic structure, cDNA, and polymorphisms C Uboldi, I Del Vecchio, MG Foti, A Azzalin, M Paulis, E Raimondi, ... Mammalian genome 16, 963-971, 2005 | 25 | 2005 |
Motor neuron degeneration, severe myopathy and TDP-43 increase in a transgenic pig model of SOD1-linked familiar ALS P Crociara, MN Chieppa, EV Costassa, E Berrone, M Gallo, ML Faro, ... Neurobiology of Disease 124, 263-275, 2019 | 24 | 2019 |
Fusion between cancer cells and macrophages occurs in a murine model of spontaneous neu+ breast cancer without increasing its metastatic potential M Lizier, A Anselmo, S Mantero, F Ficara, M Paulis, P Vezzoni, F Lucchini, ... Oncotarget 7 (38), 60793, 2016 | 20 | 2016 |
Autofluorescence properties of murine embryonic stem cells during spontaneous differentiation phases G Santin, M Paulis, P Vezzoni, G Pacchiana, G Bottiroli, AC Croce Lasers in Surgery and Medicine 45 (9), 597-607, 2013 | 20 | 2013 |
Hematopoietic cells from pluripotent stem cells: hope and promise for the treatment of inherited blood disorders I Rao, L Crisafulli, M Paulis, F Ficara Cells 11 (3), 557, 2022 | 19 | 2022 |
Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient-and cell type-specific disease phenotypes E Mangiameli, A Cecchele, F Morena, F Sanvito, V Matafora, A Cattaneo, ... Stem Cell Reports 16 (6), 1478-1495, 2021 | 15 | 2021 |
Chromosome transplantation as a novel approach for correcting complex genomic disorders M Paulis, A Castelli, L Susani, M Lizier, I Lagutina, ML Focarelli, ... Oncotarget 6 (34), 35218, 2015 | 14 | 2015 |
Chromosome transplantation: a possible approach to treat human X-linked disorders M Paulis, L Susani, A Castelli, T Suzuki, T Hara, L Straniero, S Duga, ... Molecular Therapy-Methods & Clinical Development 17, 369-377, 2020 | 13 | 2020 |
Where are we with RPE replacement therapy? A translational review from the ophthalmologist perspective R Raimondi, P Zollet, FP De Rosa, P Tsoutsanis, M Stravalaci, M Paulis, ... International Journal of Molecular Sciences 23 (2), 682, 2022 | 10 | 2022 |