112 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2020 Auricchio A, Sena-Esteves M, Gao G. Purification of Recombinant Adeno-Associated Virus 2 (rAAV2) by Heparin Column Affinity Chromatography. Cold Spring Harbor Protocols. 2020: pdb.prot095620. PMID 32358048 DOI: 10.1101/pdb.prot095620  0.76
2016 Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J, Elci OU, Chung DC, Sun J, Wright JF, Cross DR, Aravand P, Cyckowski LL, Bennicelli JL, Mingozzi F, ... Auricchio A, et al. 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 1 trial. Lancet (London, England). PMID 27375040 DOI: 10.1016/S0140-6736(16)30371-3  0.88
2016 Testa F, Maguire AM, Rossi S, Marshall K, Auricchio A, Melillo P, Bennett J, Simonelli F. Evaluation of Ocular Gene Therapy in an Italian Patient Affected by Congenital Leber Amaurosis Type 2 Treated in Both Eyes. Advances in Experimental Medicine and Biology. 854: 533-9. PMID 26427456 DOI: 10.1007/978-3-319-17121-0_71  0.88
2015 Sorrentino NC, Maffia V, Strollo S, Cacace V, Romagnoli N, Manfredi A, Ventrella D, Dondi F, Barone F, Giunti M, Graham AR, Huang Y, Kalled SL, Auricchio A, Bacci ML, et al. A comprehensive map of CNS transduction by eight recombinant adeno-associated virus serotypes upon cerebrospinal fluid administration in pigs. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 26639405 DOI: 10.1038/mt.2015.212  0.88
2015 Trapani I, Toriello E, de Simone S, Colella P, Iodice C, Polishchuk EV, Sommella A, Colecchi L, Rossi S, Simonelli F, Giunti M, Bacci ML, Polishchuk RS, Auricchio A. Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease. Human Molecular Genetics. 24: 6811-25. PMID 26420842 DOI: 10.1093/hmg/ddv386  0.88
2015 Di Donato M, Bilancio A, D'Amato L, Claudiani P, Oliviero MA, Barone MV, Auricchio A, Appella E, Migliaccio A, Auricchio F, Castoria G. CROSS TALK BETWEEN ANDROGEN RECEPTOR/ FILAMIN A AND TrkA REGULATES NEURITE OUTGROWTH IN PC12 CELLS. Molecular Biology of the Cell. PMID 26063730 DOI: 10.1091/mbc.E14-09-1352  0.88
2015 Villa F, Carrizzo A, Spinelli CC, Ferrario A, Malovini A, Maciag A, Damato A, Auricchio A, Spinetti G, Sangalli E, Dang Z, Madonna M, Ambrosio M, Sitia L, Bigini P, et al. Genetic Analysis Reveals a Longevity-Associated Protein Modulating Endothelial Function and Angiogenesis. Circulation Research. PMID 26034043 DOI: 10.1161/CIRCRESAHA.117.305875  0.88
2015 Trapani I, Banfi S, Simonelli F, Surace EM, Auricchio A. Gene therapy of inherited retinal degenerations: prospects and challenges. Human Gene Therapy. 26: 193-200. PMID 25762209 DOI: 10.1089/hum.2015.030  0.88
2015 Biferi MG, Nicoletti C, Falcone G, Puggioni EM, Passaro N, Mazzola A, Pajalunga D, Zaccagnini G, Rizzuto E, Auricchio A, Zentilin L, De Luca G, Giacca M, Martelli F, Musio A, et al. Proliferation of Multiple Cell Types in the Skeletal Muscle Tissue Elicited by Acute p21 Suppression. Molecular Therapy : the Journal of the American Society of Gene Therapy. 23: 885-95. PMID 25669433 DOI: 10.1038/mt.2015.27  0.88
2015 Ferla R, Claudiani P, Savarese M, Kozarsky K, Parini R, Scarpa M, Donati MA, Sorge G, Hopwood JJ, Parenti G, Fecarotta S, Nigro V, Sivri HS, Van Der Ploeg A, Andria G, ... ... Auricchio A, et al. Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial. Human Gene Therapy. 26: 145-52. PMID 25654180 DOI: 10.1089/hum.2014.109  0.88
2015 Auricchio A, Trapani I, Allikmets R. Gene Therapy of ABCA4-Associated Diseases. Cold Spring Harbor Perspectives in Medicine. 5: a017301. PMID 25573774 DOI: 10.1101/cshperspect.a017301  0.88
2015 Marino M, Stoilova T, Giorgi C, Bachi A, Cattaneo A, Auricchio A, Pinton P, Zito E. SEPN1, an endoplasmic reticulum-localized selenoprotein linked to skeletal muscle pathology, counteracts hyperoxidation by means of redox-regulating SERCA2 pump activity. Human Molecular Genetics. 24: 1843-55. PMID 25452428 DOI: 10.1093/hmg/ddu602  0.88
2014 Bello A, Chand A, Aviles J, Soule G, Auricchio A, Kobinger GP. Novel adeno-associated viruses derived from pig tissues transduce most major organs in mice. Scientific Reports. 4: 6644. PMID 25335510 DOI: 10.1038/srep06644  0.56
2014 Ayuso E, Blouin V, Lock M, McGorray S, Leon X, Alvira MR, Auricchio A, Bucher S, Chtarto A, Clark KR, Darmon C, Doria M, Fountain W, Gao G, Gao K, et al. Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material. Human Gene Therapy. 25: 977-87. PMID 25275822 DOI: 10.1089/hum.2014.057  0.88
2014 Trapani I, Puppo A, Auricchio A. Vector platforms for gene therapy of inherited retinopathies. Progress in Retinal and Eye Research. 43: 108-28. PMID 25124745 DOI: 10.1016/j.preteyeres.2014.08.001  0.88
2014 Puppo A, Cesi G, Marrocco E, Piccolo P, Jacca S, Shayakhmetov DM, Parks RJ, Davidson BL, Colloca S, Brunetti-Pierri N, Ng P, Donofrio G, Auricchio A. Retinal transduction profiles by high-capacity viral vectors. Gene Therapy. 21: 855-65. PMID 24989814 DOI: 10.1038/gt.2014.57  0.88
2014 Denegri M, Bongianino R, Lodola F, Boncompagni S, De Giusti VC, Avelino-Cruz JE, Liu N, Persampieri S, Curcio A, Esposito F, Pietrangelo L, Marty I, Villani L, Moyaho A, Baiardi P, ... Auricchio A, et al. Single delivery of an adeno-associated viral construct to transfer the CASQ2 gene to knock-in mice affected by catecholaminergic polymorphic ventricular tachycardia is able to cure the disease from birth to advanced age. Circulation. 129: 2673-81. PMID 24888331 DOI: 10.1161/CIRCULATIONAHA.113.006901  0.88
2014 Ferla R, Claudiani P, Cotugno G, Saccone P, De Leonibus E, Auricchio A. Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage disease. Human Gene Therapy. 25: 609-18. PMID 24725025 DOI: 10.1089/hum.2013.213  0.88
2014 Colella P, Trapani I, Cesi G, Sommella A, Manfredi A, Puppo A, Iodice C, Rossi S, Simonelli F, Giunti M, Bacci ML, Auricchio A. Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors. Gene Therapy. 21: 450-6. PMID 24572793 DOI: 10.1038/gt.2014.8  0.88
2014 Torres-Torronteras J, Viscomi C, Cabrera-Pérez R, Cámara Y, Di Meo I, Barquinero J, Auricchio A, Pizzorno G, Hirano M, Zeviani M, Martí R. Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 901-7. PMID 24448160 DOI: 10.1038/mt.2014.6  0.88
2014 Saccone P, Cotugno G, Russo F, Mastrogiacomo R, Tessitore A, Auricchio A, De Leonibus E. Sensory-motor behavioral characterization of an animal model of Maroteaux-Lamy syndrome (or Mucopolysaccharidosis VI). Scientific Reports. 4: 3644. PMID 24407717 DOI: 10.1038/srep03644  0.88
2014 Bottani E, Giordano C, Civiletto G, Di Meo I, Auricchio A, Ciusani E, Marchet S, Lamperti C, d'Amati G, Viscomi C, Zeviani M. AAV-mediated liver-specific MPV17 expression restores mtDNA levels and prevents diet-induced liver failure. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 10-7. PMID 24247928 DOI: 10.1038/mt.2013.230  0.88
2014 Trapani I, Colella P, Sommella A, Iodice C, Cesi G, de Simone S, Marrocco E, Rossi S, Giunti M, Palfi A, Farrar GJ, Polishchuk R, Auricchio A. Effective delivery of large genes to the retina by dual AAV vectors. Embo Molecular Medicine. 6: 194-211. PMID 24150896 DOI: 10.1002/emmm.201302948  0.88
2013 Doria M, Ferrara A, Auricchio A. AAV2/8 vectors purified from culture medium with a simple and rapid protocol transduce murine liver, muscle, and retina efficiently. Human Gene Therapy Methods. 24: 392-8. PMID 24116943 DOI: 10.1089/hgtb.2013.155  0.88
2013 Manfredi A, Marrocco E, Puppo A, Cesi G, Sommella A, Della Corte M, Rossi S, Giunti M, Craft CM, Bacci ML, Simonelli F, Surace EM, Auricchio A. Combined rod and cone transduction by adeno-associated virus 2/8. Human Gene Therapy. 24: 982-92. PMID 24067103 DOI: 10.1089/hum.2013.154  0.88
2013 Colella P, Sommella A, Marrocco E, Di Vicino U, Polishchuk E, Garcia Garrido M, Seeliger MW, Polishchuk R, Auricchio A. Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy. Plos One. 8: e72027. PMID 23991031 DOI: 10.1371/journal.pone.0072027  0.88
2013 Puppo A, Bello A, Manfredi A, Cesi G, Marrocco E, Della Corte M, Rossi S, Giunti M, Bacci ML, Simonelli F, Surace EM, Kobinger GP, Auricchio A. Recombinant vectors based on porcine adeno-associated viral serotypes transduce the murine and pig retina. Plos One. 8: e59025. PMID 23520549 DOI: 10.1371/journal.pone.0059025  0.88
2013 Testa F, Maguire AM, Rossi S, Pierce EA, Melillo P, Marshall K, Banfi S, Surace EM, Sun J, Acerra C, Wright JF, Wellman J, High KA, Auricchio A, Bennett J, et al. Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Ophthalmology. 120: 1283-91. PMID 23474247 DOI: 10.1016/j.ophtha.2012.11.048  0.88
2013 Colella P, Auricchio A. Photoreceptor degeneration in mice: adeno-associated viral vector-mediated delivery of erythropoietin. Methods in Molecular Biology (Clifton, N.J.). 982: 237-63. PMID 23456874 DOI: 10.1007/978-1-62703-308-4_16  0.88
2013 Rotundo IL, Lancioni A, Savarese M, D'Orsi L, Iacomino M, Nigro G, Piluso G, Auricchio A, Nigro V. Use of a lower dosage liver-detargeted AAV vector to prevent hamster muscular dystrophy. Human Gene Therapy. 24: 424-30. PMID 23427808 DOI: 10.1089/hum.2012.121  0.88
2013 Ferla R, O'Malley T, Calcedo R, O'Donnell P, Wang P, Cotugno G, Claudiani P, Wilson JM, Haskins M, Auricchio A. Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8. Human Gene Therapy. 24: 163-9. PMID 23194248 DOI: 10.1089/hum.2012.179  0.88
2013 Bartolomeo R, Polishchuk EV, Volpi N, Polishchuk RS, Auricchio A. Pharmacological read-through of nonsense ARSB mutations as a potential therapeutic approach for mucopolysaccharidosis VI. Journal of Inherited Metabolic Disease. 36: 363-71. PMID 22971959 DOI: 10.1007/s10545-012-9521-y  0.88
2012 Di Meo I, Auricchio A, Lamperti C, Burlina A, Viscomi C, Zeviani M. Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy. Embo Molecular Medicine. 4: 1008-14. PMID 22903887 DOI: 10.1002/emmm.201201433  0.88
2012 Pastore N, Nusco E, Vaníkova J, Sepe RM, Vetrini F, McDonagh A, Auricchio A, Vitek L, Brunetti-Pierri N. Sustained reduction of hyperbilirubinemia in Gunn rats after adeno-associated virus-mediated gene transfer of bilirubin UDP-glucuronosyltransferase isozyme 1A1 to skeletal muscle. Human Gene Therapy. 23: 1082-9. PMID 22765254 DOI: 10.1089/hum.2012.018  0.88
2012 Colella P, Auricchio A. Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients. Human Gene Therapy. 23: 796-807. PMID 22734691 DOI: 10.1089/hum.2012.123  0.88
2012 Testa F, Rossi S, Sodi A, Passerini I, Di Iorio V, Della Corte M, Banfi S, Surace EM, Menchini U, Auricchio A, Simonelli F. Correlation between photoreceptor layer integrity and visual function in patients with Stargardt disease: implications for gene therapy. Investigative Ophthalmology & Visual Science. 53: 4409-15. PMID 22661472 DOI: 10.1167/iovs.11-8201  0.88
2012 Cotugno G, Annunziata P, Barone MV, Karali M, Banfi S, Auricchio A. Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction. Plos One. 7: e33286. PMID 22428010 DOI: 10.1371/journal.pone.0033286  0.88
2012 Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, McCague S, Pierce EA, Chen Y, Bennicelli JL, Zhu X, Ying GS, Sun J, Wright JF, Auricchio A, et al. AAV2 gene therapy readministration in three adults with congenital blindness. Science Translational Medicine. 4: 120ra15. PMID 22323828 DOI: 10.1126/scitranslmed.3002865  0.88
2012 Denegri M, Avelino-Cruz JE, Boncompagni S, De Simone SA, Auricchio A, Villani L, Volpe P, Protasi F, Napolitano C, Priori SG. Viral gene transfer rescues arrhythmogenic phenotype and ultrastructural abnormalities in adult calsequestrin-null mice with inherited arrhythmias. Circulation Research. 110: 663-8. PMID 22298808 DOI: 10.1161/CIRCRESAHA.111.263939  0.88
2012 Vandenberghe LH, Auricchio A. Novel adeno-associated viral vectors for retinal gene therapy. Gene Therapy. 19: 162-8. PMID 21993172 DOI: 10.1038/gt.2011.151  0.88
2011 Auricchio A. Fighting blindness with adeno-associated virus serotype 8. Human Gene Therapy. 22: 1169-70. PMID 22044092 DOI: 10.1089/hum.2011.2521  0.88
2011 Rotundo IL, Faraso S, De Leonibus E, Nigro G, Vitiello C, Lancioni A, Di Napoli D, Castaldo S, Russo V, Russo F, Piluso G, Auricchio A, Nigro V. Worsening of cardiomyopathy using deflazacort in an animal model rescued by gene therapy. Plos One. 6: e24729. PMID 21931833 DOI: 10.1371/journal.pone.0024729  0.88
2011 Karali M, Manfredi A, Puppo A, Marrocco E, Gargiulo A, Allocca M, Corte MD, Rossi S, Giunti M, Bacci ML, Simonelli F, Surace EM, Banfi S, Auricchio A. MicroRNA-restricted transgene expression in the retina. Plos One. 6: e22166. PMID 21818300 DOI: 10.1371/journal.pone.0022166  0.88
2011 Ashtari M, Cyckowski LL, Monroe JF, Marshall KA, Chung DC, Auricchio A, Simonelli F, Leroy BP, Maguire AM, Shindler KS, Bennett J. The human visual cortex responds to gene therapy-mediated recovery of retinal function. The Journal of Clinical Investigation. 121: 2160-8. PMID 21606598 DOI: 10.1172/JCI57377  0.88
2011 Testa F, Surace EM, Rossi S, Marrocco E, Gargiulo A, Di Iorio V, Ziviello C, Nesti A, Fecarotta S, Bacci ML, Giunti M, Della Corte M, Banfi S, Auricchio A, Simonelli F. Evaluation of Italian patients with leber congenital amaurosis due to AIPL1 mutations highlights the potential applicability of gene therapy. Investigative Ophthalmology & Visual Science. 52: 5618-24. PMID 21474771 DOI: 10.1167/iovs.10-6543  0.88
2011 Colella P, Iodice C, Di Vicino U, Annunziata I, Surace EM, Auricchio A. Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration. Human Molecular Genetics. 20: 2251-62. PMID 21421996 DOI: 10.1093/hmg/ddr115  0.88
2011 Mussolino C, della Corte M, Rossi S, Viola F, Di Vicino U, Marrocco E, Neglia S, Doria M, Testa F, Giovannoni R, Crasta M, Giunti M, Villani E, Lavitrano M, Bacci ML, ... ... Auricchio A, et al. AAV-mediated photoreceptor transduction of the pig cone-enriched retina. Gene Therapy. 18: 637-45. PMID 21412286 DOI: 10.1038/gt.2011.3  0.88
2011 Spampanato C, De Leonibus E, Dama P, Gargiulo A, Fraldi A, Sorrentino NC, Russo F, Nusco E, Auricchio A, Surace EM, Ballabio A. Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 860-9. PMID 21326216 DOI: 10.1038/mt.2010.299  0.88
2011 Allocca M, Manfredi A, Iodice C, Di Vicino U, Auricchio A. AAV-mediated gene replacement, either alone or in combination with physical and pharmacological agents, results in partial and transient protection from photoreceptor degeneration associated with betaPDE deficiency. Investigative Ophthalmology & Visual Science. 52: 5713-9. PMID 21273543 DOI: 10.1167/iovs.10-6269  0.88
2011 Mussolino C, Sanges D, Marrocco E, Bonetti C, Di Vicino U, Marigo V, Auricchio A, Meroni G, Surace EM. Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa. Embo Molecular Medicine. 3: 118-28. PMID 21268285 DOI: 10.1002/emmm.201000119  0.88
2011 Cotugno G, Annunziata P, Tessitore A, O'Malley T, Capalbo A, Faella A, Bartolomeo R, O'Donnell P, Wang P, Russo F, Sleeper MM, Knox VW, Fernandez S, Levanduski L, Hopwood J, ... ... Auricchio A, et al. Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 461-9. PMID 21119624 DOI: 10.1038/mt.2010.257  0.88
2011 Cotugno G, Aurilio M, Annunziata P, Capalbo A, Faella A, Rinaldi V, Strisciuglio C, Di Tommaso M, Aloj L, Auricchio A. Noninvasive repetitive imaging of somatostatin receptor 2 gene transfer with positron emission tomography. Human Gene Therapy. 22: 189-96. PMID 20825281 DOI: 10.1089/hum.2010.098  0.88
2011 Ashtari M, Cyckowski LL, Monroe JF, Marshall KA, Chung DC, Auricchio A, Simonelli F, Leroy BP, Maguire AM, Shindler KS, Bennett J. The human visual cortex responds to gene therapy-mediated recovery of retinal function (The Journal of Clinical Investigation (2011) 121, 6, (2160-2168) DOI: 10.1172/JCI57377) Journal of Clinical Investigation. 121: 2945. DOI: 10.1172/JCI59208  0.88
2010 Cacchiarelli D, Martone J, Girardi E, Cesana M, Incitti T, Morlando M, Nicoletti C, Santini T, Sthandier O, Barberi L, Auricchio A, Musarò A, Bozzoni I. MicroRNAs involved in molecular circuitries relevant for the Duchenne muscular dystrophy pathogenesis are controlled by the dystrophin/nNOS pathway. Cell Metabolism. 12: 341-51. PMID 20727829 DOI: 10.1016/j.cmet.2010.07.008  0.88
2010 Colella P, Auricchio A. AAV-mediated gene supply for treatment of degenerative and neovascular retinal diseases. Current Gene Therapy. 10: 371-80. PMID 20712580  0.88
2010 Lock M, McGorray S, Auricchio A, Ayuso E, Beecham EJ, Blouin-Tavel V, Bosch F, Bose M, Byrne BJ, Caton T, Chiorini JA, Chtarto A, Clark KR, Conlon T, Darmon C, et al. Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Human Gene Therapy. 21: 1273-85. PMID 20486768 DOI: 10.1089/hum.2009.223  0.88
2010 Cotugno G, Tessitore A, Capalbo A, Annunziata P, Strisciuglio C, Faella A, Aurilio M, Di Tommaso M, Russo F, Mancini A, De Leonibus E, Aloj L, Auricchio A. Different serum enzyme levels are required to rescue the various systemic features of the mucopolysaccharidoses. Human Gene Therapy. 21: 555-69. PMID 20021231 DOI: 10.1089/hum.2009.189  0.88
2010 Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, Rossi S, Marshall K, Banfi S, Surace EM, Sun J, Redmond TM, Zhu X, Shindler KS, Ying GS, ... ... Auricchio A, et al. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 643-50. PMID 19953081 DOI: 10.1038/mt.2009.277  0.88
2010 Ponder KP, Auricchio A. Gene therapy for ocular problems in mucopolysaccharidosis: An experimental and promising approach with benefits in animal models - a review Clinical and Experimental Ophthalmology. 38: 43-51. DOI: 10.1111/j.1442-9071.2010.02367.x  0.88
2009 Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JI, et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet. 374: 1597-605. PMID 19854499 DOI: 10.1016/S0140-6736(09)61836-5  0.88
2009 Bello A, Tran K, Chand A, Doria M, Allocca M, Hildinger M, Beniac D, Kranendonk C, Auricchio A, Kobinger GP. Isolation and evaluation of novel adeno-associated virus sequences from porcine tissues Gene Therapy. 16: 1320-1328. PMID 19626054 DOI: 10.1038/gt.2009.82  0.88
2009 Gargiulo A, Bonetti C, Montefusco S, Neglia S, Di Vicino U, Marrocco E, Corte MD, Domenici L, Auricchio A, Surace EM. AAV-mediated tyrosinase gene transfer restores melanogenesis and retinal function in a model of oculo-cutaneous albinism type I (OCA1). Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1347-54. PMID 19436266 DOI: 10.1038/mt.2009.112  0.88
2009 Vitiello C, Faraso S, Sorrentino NC, Di Salvo G, Nusco E, Nigro G, Cutillo L, Calabrò R, Auricchio A, Nigro V. Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatments. Plos One. 4: e5051. PMID 19333401 DOI: 10.1371/journal.pone.0005051  0.88
2009 Colella P, Cotugno G, Auricchio A. Ocular gene therapy: current progress and future prospects. Trends in Molecular Medicine. 15: 23-31. PMID 19097940 DOI: 10.1016/j.molmed.2008.11.003  0.88
2008 Denti MA, Incitti T, Sthandier O, Nicoletti C, De Angelis FG, Rizzuto E, Auricchio A, Musarò A, Bozzoni I. Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice. Human Gene Therapy. 19: 601-8. PMID 18500943 DOI: 10.1089/hum.2008.012  0.88
2008 Maguire AM, Simonelli F, Pierce EA, Pugh EN, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, ... ... Auricchio A, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. The New England Journal of Medicine. 358: 2240-8. PMID 18441370 DOI: 10.1056/NEJMoa0802315  0.88
2008 Allocca M, Doria M, Petrillo M, Colella P, Garcia-Hoyos M, Gibbs D, Kim SR, Maguire A, Rex TS, Di Vicino U, Cutillo L, Sparrow JR, Williams DS, Bennett J, Auricchio A. Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. The Journal of Clinical Investigation. 118: 1955-64. PMID 18414684 DOI: 10.1172/JCI34316  0.88
2008 Tessitore A, Faella A, O'Malley T, Cotugno G, Doria M, Kunieda T, Matarese G, Haskins M, Auricchio A. Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 30-7. PMID 17955027 DOI: 10.1038/sj.mt.6300325  0.88
2008 Surace EM, Auricchio A. Versatility of AAV vectors for retinal gene transfer. Vision Research. 48: 353-9. PMID 17923143 DOI: 10.1016/j.visres.2007.07.027  0.88
2007 Allocca M, Di Vicino U, Petrillo M, Carlomagno F, Domenici L, Auricchio A. Constitutive and AP20187-induced Ret activation in photoreceptors does not protect from light-induced damage. Investigative Ophthalmology & Visual Science. 48: 5199-206. PMID 17962474 DOI: 10.1167/iovs.07-0140  0.88
2007 Fraldi A, Hemsley K, Crawley A, Lombardi A, Lau A, Sutherland L, Auricchio A, Ballabio A, Hopwood JJ. Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes. Human Molecular Genetics. 16: 2693-702. PMID 17725987 DOI: 10.1093/hmg/ddm223  0.88
2007 Simonelli F, Ziviello C, Testa F, Rossi S, Fazzi E, Bianchi PE, Fossarello M, Signorini S, Bertone C, Galantuomo S, Brancati F, Valente EM, Ciccodicola A, Rinaldi E, Auricchio A, et al. Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients. Investigative Ophthalmology & Visual Science. 48: 4284-90. PMID 17724218 DOI: 10.1167/iovs.07-0068  0.88
2007 Allocca M, Mussolino C, Garcia-Hoyos M, Sanges D, Iodice C, Petrillo M, Vandenberghe LH, Wilson JM, Marigo V, Surace EM, Auricchio A. Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. Journal of Virology. 81: 11372-80. PMID 17699581 DOI: 10.1128/JVI.01327-07  0.88
2007 O'Reilly M, Palfi A, Chadderton N, Millington-Ward S, Ader M, Cronin T, Tuohy T, Auricchio A, Hildinger M, Tivnan A, McNally N, Humphries MM, Kiang AS, Humphries P, Kenna PF, et al. RNA interference-mediated suppression and replacement of human rhodopsin in vivo. American Journal of Human Genetics. 81: 127-35. PMID 17564969 DOI: 10.1086/519025  0.88
2007 Gargiulo A, Auricchio R, Barone MV, Cotugno G, Reardon W, Milla PJ, Ballabio A, Ciccodicola A, Auricchio A. Filamin A is mutated in X-linked chronic idiopathic intestinal pseudo-obstruction with central nervous system involvement. American Journal of Human Genetics. 80: 751-8. PMID 17357080 DOI: 10.1086/513321  0.88
2007 Cotugno G, Formisano P, Giacco F, Colella P, Beguinot F, Auricchio A. AP20187-mediated activation of a chimeric insulin receptor results in insulin-like actions in skeletal muscle and liver of diabetic mice. Human Gene Therapy. 18: 106-17. PMID 17328681 DOI: 10.1089/hum.2006.116  0.88
2007 Fraldi A, Biffi A, Lombardi A, Visigalli I, Pepe S, Settembre C, Nusco E, Auricchio A, Naldini L, Ballabio A, Cosma MP. SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies. The Biochemical Journal. 403: 305-12. PMID 17206939 DOI: 10.1042/BJ20061783  0.88
2006 Allocca M, Tessitore A, Cotugno G, Auricchio A. AAV-mediated gene transfer for retinal diseases. Expert Opinion On Biological Therapy. 6: 1279-94. PMID 17223737 DOI: 10.1517/14712598.6.12.1279  0.88
2006 Tessitore A, Parisi F, Denti MA, Allocca M, Di Vicino U, Domenici L, Bozzoni I, Auricchio A. Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model. Molecular Therapy : the Journal of the American Society of Gene Therapy. 14: 692-9. PMID 16979943 DOI: 10.1016/j.ymthe.2006.07.008  0.88
2006 Denti MA, Rosa A, D'Antona G, Sthandier O, De Angelis FG, Nicoletti C, Allocca M, Pansarasa O, Parente V, Musarò A, Auricchio A, Bottinelli R, Bozzoni I. Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice. Human Gene Therapy. 17: 565-74. PMID 16716113 DOI: 10.1089/hum.2006.17.565  0.88
2006 Vetrini F, Tammaro R, Bondanza S, Surace EM, Auricchio A, De Luca M, Ballabio A, Marigo V. Aberrant splicing in the ocular albinism type 1 gene (OA1/GPR143) is corrected in vitro by morpholino antisense oligonucleotides. Human Mutation. 27: 420-6. PMID 16550551 DOI: 10.1002/humu.20303  0.88
2006 Cardone M, Polito VA, Pepe S, Mann L, D'Azzo A, Auricchio A, Ballabio A, Cosma MP. Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery. Human Molecular Genetics. 15: 1225-36. PMID 16505002 DOI: 10.1093/hmg/ddl038  0.88
2006 Denti MA, Rosa A, D'Antona G, Sthandier O, De Angelis FG, Nicoletti C, Allocca M, Pansarasa O, Parente V, Musarò A, Auricchio A, Bottinelli R, Bozzoni I. Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model. Proceedings of the National Academy of Sciences of the United States of America. 103: 3758-63. PMID 16501048 DOI: 10.1073/pnas.0508917103  0.88
2006 Pirozzi M, Quattrini A, Andolfi G, Dina G, Malaguti MC, Auricchio A, Rugarli EI. Intramuscular viral delivery of paraplegin rescues peripheral axonopathy in a model of hereditary spastic paraplegia. The Journal of Clinical Investigation. 116: 202-8. PMID 16357941 DOI: 10.1172/JCI26210  0.88
2006 Surace EM, Balaggan KS, Tessitore A, Mussolino C, Cotugno G, Bonetti C, Vitale A, Ali RR, Auricchio A. Inhibition of ocular neovascularization by hedgehog blockade. Molecular Therapy : the Journal of the American Society of Gene Therapy. 13: 573-9. PMID 16343995 DOI: 10.1016/j.ymthe.2005.10.010  0.88
2005 Surace EM, Domenici L, Cortese K, Cotugno G, Di Vicino U, Venturi C, Cellerino A, Marigo V, Tacchetti C, Ballabio A, Auricchio A. Amelioration of both functional and morphological abnormalities in the retina of a mouse model of ocular albinism following AAV-mediated gene transfer. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 652-8. PMID 16023414 DOI: 10.1016/j.ymthe.2005.06.001  0.88
2005 Auricchio A, Rolling F. Adeno-associated viral vectors for retinal gene transfer and treatment of retinal diseases. Current Gene Therapy. 5: 339-48. PMID 15975011 DOI: 10.2174/1566523054065020  0.88
2005 Lebherz C, Maguire AM, Auricchio A, Tang W, Aleman TS, Wei Z, Grant R, Cideciyan AV, Jacobson SG, Wilson JM, Bennett J. Nonhuman primate models for diabetic ocular neovascularization using AAV2-mediated overexpression of vascular endothelial growth factor. Diabetes. 54: 1141-9. PMID 15793254 DOI: 10.2337/diabetes.54.4.1141  0.88
2005 Lebherz C, Auricchio A, Maguire AM, Rivera VM, Tang W, Grant RL, Clackson T, Bennett J, Wilson JM. Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates. Human Gene Therapy. 16: 178-86. PMID 15761258 DOI: 10.1089/hum.2005.16.178  0.88
2005 Alfano G, Vitiello C, Caccioppoli C, Caramico T, Carola A, Szego MJ, McInnes RR, Auricchio A, Banfi S. Natural antisense transcripts associated with genes involved in eye development. Human Molecular Genetics. 14: 913-23. PMID 15703187 DOI: 10.1093/hmg/ddi084  0.88
2004 Cotugno G, Pollock R, Formisano P, Linher K, Beguinot F, Auricchio A. Pharmacological regulation of the insulin receptor signaling pathway mimics insulin action in cells transduced with viral vectors. Human Gene Therapy. 15: 1101-8. PMID 15610610 DOI: 10.1089/hum.2004.15.1101  0.88
2004 Wong WT, Rex TS, Auricchio A, Maguire AM, Chung D, Tang W, Bennett J. Effect of over-expression of pigment epithelium derived factor (PEDF) on developing retinal vasculature in the mouse. Molecular Vision. 10: 837-44. PMID 15547489  0.88
2004 Rex TS, Allocca M, Domenici L, Surace EM, Maguire AM, Lyubarsky A, Cellerino A, Bennett J, Auricchio A. Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration. Molecular Therapy : the Journal of the American Society of Gene Therapy. 10: 855-61. PMID 15509503 DOI: 10.1016/j.ymthe.2004.07.027  0.88
2004 Vetrini F, Auricchio A, Du J, Angeletti B, Fisher DE, Ballabio A, Marigo V. The microphthalmia transcription factor (Mitf) controls expression of the ocular albinism type 1 gene: link between melanin synthesis and melanosome biogenesis. Molecular and Cellular Biology. 24: 6550-9. PMID 15254223 DOI: 10.1128/MCB.24.15.6550-6559.2004  0.88
2004 Ferreirinha F, Quattrini A, Pirozzi M, Valsecchi V, Dina G, Broccoli V, Auricchio A, Piemonte F, Tozzi G, Gaeta L, Casari G, Ballabio A, Rugarli EI. Axonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transport. The Journal of Clinical Investigation. 113: 231-42. PMID 14722615 DOI: 10.1172/JCI20138  0.88
2004 Hildinger M, Auricchio A. Advances in AAV-mediated gene transfer for the treatment of inherited disorders. European Journal of Human Genetics : Ejhg. 12: 263-71. PMID 14722585 DOI: 10.1038/sj.ejhg.5201153  0.88
2004 Croyle MA, Callahan SM, Auricchio A, Schumer G, Linse KD, Wilson JM, Brunner LJ, Kobinger GP. PEGylation of a vesicular stomatitis virus G pseudotyped lentivirus vector prevents inactivation in serum. Journal of Virology. 78: 912-21. PMID 14694122 DOI: 10.1128/JVI.78.2.912-921.2004  0.88
2003 Surace EM, Auricchio A. Adeno-associated viral vectors for retinal gene transfer. Progress in Retinal and Eye Research. 22: 705-19. PMID 14575721 DOI: 10.1016/S1350-9462(03)00052-1  0.88
2003 Surace EM, Auricchio A, Reich SJ, Rex T, Glover E, Pineles S, Tang W, O'Connor E, Lyubarsky A, Savchenko A, Pugh EN, Maguire AM, Wilson JM, Bennett J. Delivery of adeno-associated virus vectors to the fetal retina: impact of viral capsid proteins on retinal neuronal progenitor transduction. Journal of Virology. 77: 7957-63. PMID 12829835 DOI: 10.1128/JVI.77.14.7957-7963.2003  0.88
2003 Auricchio A, Acton PD, Hildinger M, Louboutin JP, Plössl K, O'Connor E, Kung HF, Wilson JM. In vivo quantitative noninvasive imaging of gene transfer by single-photon emission computerized tomography. Human Gene Therapy. 14: 255-61. PMID 12639305 DOI: 10.1089/10430340360535805  0.76
2003 Reich SJ, Auricchio A, Hildinger M, Glover E, Maguire AM, Wilson JM, Bennett J. Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy. Human Gene Therapy. 14: 37-44. PMID 12573057 DOI: 10.1089/10430340360464697  0.76
2002 Auricchio A, Behling KC, Maguire AM, O'Connor EM, Bennett J, Wilson JM, Tolentino MJ. Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents. Molecular Therapy : the Journal of the American Society of Gene Therapy. 6: 490-4. PMID 12377190 DOI: 10.1006/mthe.2002.0702  0.76
2002 Auricchio A, O'Connor E, Weiner D, Gao GP, Hildinger M, Wang L, Calcedo R, Wilson JM. Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. The Journal of Clinical Investigation. 110: 499-504. PMID 12189244 DOI: 10.1172/JCI15780  0.88
2002 Auricchio A, Rivera VM, Clackson T, O'Connor EE, Maguire AM, Tolentino MJ, Bennett J, Wilson JM. Pharmacological regulation of protein expression from adeno-associated viral vectors in the eye. Molecular Therapy : the Journal of the American Society of Gene Therapy. 6: 238-42. PMID 12161190  0.76
2002 Auricchio A, Gao GP, Yu QC, Raper S, Rivera VM, Clackson T, Wilson JM. Constitutive and regulated expression of processed insulin following in vivo hepatic gene transfer. Gene Therapy. 9: 963-71. PMID 12085245 DOI: 10.1038/sj.gt.3301746  0.76
2001 Auricchio A, Kobinger G, Anand V, Hildinger M, O'Connor E, Maguire AM, Wilson JM, Bennett J. Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Human Molecular Genetics. 10: 3075-81. PMID 11751689  0.76
2001 Shifrin AL, Auricchio A, Yu QC, Wilson J, Raper SE. Adenoviral vector-mediated insulin gene transfer in the mouse pancreas corrects streptozotocin-induced hyperglycemia. Gene Therapy. 8: 1480-9. PMID 11593361 DOI: 10.1038/sj.gt.3301544  0.44
2001 Auricchio A, O'Connor E, Hildinger M, Wilson JM. A single-step affinity column for purification of serotype-5 based adeno-associated viral vectors. Molecular Therapy : the Journal of the American Society of Gene Therapy. 4: 372-4. PMID 11592841 DOI: 10.1006/mthe.2001.0462  0.76
2001 Hildinger M, Auricchio A, Gao G, Wang L, Chirmule N, Wilson JM. Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer. Journal of Virology. 75: 6199-203. PMID 11390622 DOI: 10.1128/JVI.75.13.6199-6203.2001  0.76
2001 Dejneka NS, Auricchio A, Maguire AM, Ye X, Gao GP, Wilson JM, Bennett J. Pharmacologically regulated gene expression in the retina following transduction with viral vectors. Gene Therapy. 8: 442-6. PMID 11313822 DOI: 10.1038/sj.gt.3301413  0.76
2001 Auricchio A, Zhou R, Wilson JM, Glickson JD. In vivo detection of gene expression in liver by 31P nuclear magnetic resonance spectroscopy employing creatine kinase as a marker gene. Proceedings of the National Academy of Sciences of the United States of America. 98: 5205-10. PMID 11296261 DOI: 10.1073/pnas.081508598  0.88
2001 Auricchio A, Hildinger M, O'Connor E, Gao GP, Wilson JM. Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column. Human Gene Therapy. 12: 71-6. PMID 11177544 DOI: 10.1089/104303401450988  0.76
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