| TFEB links autophagy to lysosomal biogenesis C Settembre, C Di Malta, VA Polito, MG Arencibia, F Vetrini, S Erdin, ... science 332 (6036), 1429-1433, 2011 | 2990 | 2011 |
| Emerging issues in AAV-mediated in vivo gene therapy P Colella, G Ronzitti, F Mingozzi Molecular Therapy-Methods & Clinical Development 8, 87-104, 2018 | 789 | 2018 |
| Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice M Allocca, M Doria, M Petrillo, P Colella, M Garcia-Hoyos, D Gibbs, ... The Journal of clinical investigation 118 (5), 1955-1964, 2008 | 352 | 2008 |
| Effective delivery of large genes to the retina by dual AAV vectors I Trapani, P Colella, A Sommella, C Iodice, G Cesi, S de Simone, ... EMBO molecular medicine 6 (2), 194-211, 2014 | 258 | 2014 |
| Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase F Puzzo, P Colella, MG Biferi, D Bali, NK Paulk, P Vidal, F Collaud, ... Science translational medicine 9 (418), eaam6375, 2017 | 119 | 2017 |
| Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors P Colella, I Trapani, G Cesi, A Sommella, A Manfredi, A Puppo, C Iodice, ... Gene therapy 21 (4), 450-456, 2014 | 115 | 2014 |
| Ocular gene therapy: current progress and future prospects P Colella, G Cotugno, A Auricchio Trends in molecular medicine 15 (1), 23-31, 2009 | 101 | 2009 |
| Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease I Trapani, E Toriello, S De Simone, P Colella, C Iodice, EV Polishchuk, ... Human molecular genetics 24 (23), 6811-6825, 2015 | 96 | 2015 |
| AAV gene transfer with tandem promoter design prevents anti-transgene immunity and provides persistent efficacy in neonate pompe mice P Colella, P Sellier, HC Verdera, F Puzzo, L van Wittenberghe, ... Molecular Therapy-Methods & Clinical Development 12, 85-101, 2019 | 70 | 2019 |
| Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients P Colella, A Auricchio Human gene therapy 23 (8), 796-807, 2012 | 64 | 2012 |
| Effective delivery of large genes by dual aav vectors A Auricchio, P Colella, I Trapani | 51 | 2016 |
| Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration P Colella, C Iodice, U Di Vicino, I Annunziata, EM Surace, A Auricchio Human molecular genetics 20 (11), 2251-2262, 2011 | 46 | 2011 |
| Engineering monocyte/macrophage− specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing SG Scharenberg, E Poletto, KL Lucot, P Colella, A Sheikali, TJ Montine, ... Nature Communications 11 (1), 3327, 2020 | 41 | 2020 |
| Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy P Colella, A Sommella, E Marrocco, U Di Vicino, E Polishchuk, ... PloS one 8 (8), e72027, 2013 | 37 | 2013 |
| Rescue of GSDIII phenotype with gene transfer requires liver-and muscle-targeted GDE expression P Vidal, S Pagliarani, P Colella, HC Verdera, L Jauze, M Gjorgjieva, ... Molecular Therapy 26 (3), 890-901, 2018 | 32 | 2018 |
| Gene therapy for Pompe disease: the time is now P Colella, F Mingozzi Human Gene Therapy 30 (10), 1245-1262, 2019 | 31 | 2019 |
| AAV-mediated gene supply for treatment of degenerative and neovascular retinal diseases P Colella, A Auricchio Current gene therapy 10 (5), 371-380, 2010 | 30 | 2010 |
| Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients E Masat, P Laforêt, M De Antonio, G Corre, B Perniconi, N Taouagh, ... Scientific reports 6 (1), 36182, 2016 | 29 | 2016 |
| Role of regulatory T cell and effector T cell exhaustion in liver-mediated transgene tolerance in muscle J Poupiot, HC Verdera, R Hardet, P Colella, F Collaud, L Bartolo, ... Molecular Therapy-Methods & Clinical Development 15, 83-100, 2019 | 22 | 2019 |
| Rescue of advanced Pompe disease in mice with hepatic expression of secretable acid α-glucosidase U Cagin, F Puzzo, MJ Gomez, M Moya-Nilges, P Sellier, C Abad, ... Molecular Therapy 28 (9), 2056-2072, 2020 | 19 | 2020 |
