| Safety and efficacy of gene transfer for Leber's congenital amaurosis AM Maguire, F Simonelli, EA Pierce, EN Pugh Jr, F Mingozzi, J Bennicelli, ... New England Journal of Medicine 358 (21), 2240-2248, 2008 | 2576 | 2008 |
| Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial AM Maguire, KA High, A Auricchio, JF Wright, EA Pierce, F Testa, ... The Lancet 374 (9701), 1597-1605, 2009 | 1037 | 2009 |
| Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration F Simonelli, AM Maguire, F Testa, EA Pierce, F Mingozzi, JL Bennicelli, ... Molecular Therapy 18 (3), 643-650, 2010 | 721 | 2010 |
| Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase … J Bennett, J Wellman, KA Marshall, S McCague, M Ashtari, ... The Lancet 388 (10045), 661-672, 2016 | 486 | 2016 |
| AAV2 gene therapy readministration in three adults with congenital blindness J Bennett, M Ashtari, J Wellman, KA Marshall, LL Cyckowski, DC Chung, ... Science translational medicine 4 (120), 120ra15-120ra15, 2012 | 470 | 2012 |
| Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model A Auricchio, G Kobinger, V Anand, M Hildinger, E O'Connor, AM Maguire, ... Human molecular genetics 10 (26), 3075-3081, 2001 | 460 | 2001 |
| Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column A Auricchio, M Hildinger, E O'Connor, GP Gao, JM Wilson Human gene therapy 12 (1), 71-76, 2001 | 422 | 2001 |
| Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2 F Testa, AM Maguire, S Rossi, EA Pierce, P Melillo, K Marshall, S Banfi, ... Ophthalmology 120 (6), 1283-1291, 2013 | 403 | 2013 |
| Axonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transport F Ferreirinha, A Quattrini, M Pirozzi, V Valsecchi, G Dina, V Broccoli, ... The Journal of clinical investigation 113 (2), 231-242, 2004 | 376 | 2004 |
| 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 | 354 | 2008 |
| Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors M Allocca, C Mussolino, M Garcia-Hoyos, D Sanges, C Iodice, M Petrillo, ... Journal of virology 81 (20), 11372-11380, 2007 | 309 | 2007 |
| MicroRNAs involved in molecular circuitries relevant for the Duchenne muscular dystrophy pathogenesis are controlled by the dystrophin/nNOS pathway D Cacchiarelli, J Martone, E Girardi, M Cesana, T Incitti, M Morlando, ... Cell metabolism 12 (4), 341-351, 2010 | 275 | 2010 |
| Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer M Hildinger, A Auricchio, G Gao, L Wang, N Chirmule, JM Wilson Journal of virology 75 (13), 6199-6203, 2001 | 264 | 2001 |
| 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 | 260 | 2014 |
| RNA interference–mediated suppression and replacement of human rhodopsin in vivo M O’Reilly, A Palfi, N Chadderton, S Millington-Ward, M Ader, T Cronin, ... The American Journal of Human Genetics 81 (1), 127-135, 2007 | 223 | 2007 |
| Endothelin-B receptor mutations in patients with isolated Hirschsprung disease from a non-inbred population A Auricchio, G Casari, A Staiano, A Ballabio Human molecular genetics 5 (3), 351-354, 1996 | 208 | 1996 |
| Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents A Auricchio, KC Behling, AM Maguire, EE O'connor, J Bennett, JM Wilson, ... Molecular therapy 6 (4), 490-494, 2002 | 199 | 2002 |
| Vector platforms for gene therapy of inherited retinopathies I Trapani, A Puppo, A Auricchio Progress in retinal and eye research 43, 108-128, 2014 | 194 | 2014 |
| Versatility of AAV vectors for retinal gene transfer EM Surace, A Auricchio Vision research 48 (3), 353-359, 2008 | 193 | 2008 |
| PEGylation of a vesicular stomatitis virus G pseudotyped lentivirus vector prevents inactivation in serum MA Croyle, SM Callahan, A Auricchio, G Schumer, KD Linse, JM Wilson, ... Journal of virology 78 (2), 912-921, 2004 | 177 | 2004 |
