| Year |
Citation |
Score |
| 2023 |
Bueren JA, Auricchio A. Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases. Human Gene Therapy. 34: 763-775. PMID 37694572 DOI: 10.1089/hum.2023.152 |
0.32 |
|
| 2023 |
Ferla R, Dell'Aquila F, Doria M, Ferraiuolo M, Noto A, Grazioli F, Ammendola V, Testa F, Melillo P, Iodice C, Risca G, Tedesco N, le Brun PR, Surace EM, Simonelli F, ... ... Auricchio A, et al. Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B. Molecular Therapy. Methods & Clinical Development. 28: 396-411. PMID 36910588 DOI: 10.1016/j.omtm.2023.02.002 |
0.362 |
|
| 2023 |
Boffa I, Polishchuk E, De Stefano L, Dell'Aquila F, Nusco E, Marrocco E, Audano M, Pedretti S, Caterino M, Bellezza I, Ruoppolo M, Mitro N, Cellini B, Auricchio A, Brunetti-Pierri N. Liver-directed gene therapy for ornithine aminotransferase deficiency. Embo Molecular Medicine. e17033. PMID 36647689 DOI: 10.15252/emmm.202217033 |
0.399 |
|
| 2022 |
Brunetti-Pierri N, Ferla R, Ginocchio VM, Rossi A, Fecarotta S, Romano R, Parenti G, Yildiz Y, Zancan S, Pecorella V, Dell'Anno M, Graziano M, Alliegro M, Andria G, Santamaria F, ... ... Auricchio A, et al. Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI. Nejm Evidence. 1: EVIDoa2200052. PMID 38319253 DOI: 10.1056/EVIDoa2200052 |
0.305 |
|
| 2022 |
Padula A, Petruzzelli R, Philbert SA, Church SJ, Esposito F, Campione S, Monti M, Capolongo F, Perna C, Nusco E, Schmidt HH, Auricchio A, Cooper GJS, Polishchuk R, Piccolo P. Full-length ATP7B reconstituted through protein -splicing corrects Wilson disease in mice. Molecular Therapy. Methods & Clinical Development. 26: 495-504. PMID 36092366 DOI: 10.1016/j.omtm.2022.08.004 |
0.315 |
|
| 2022 |
Tornabene P, Ferla R, Llado-Santaeularia M, Centrulo M, Dell'Anno M, Esposito F, Marrocco E, Pone E, Minopoli R, Iodice C, Nusco E, Rossi S, Lyubenova H, Manfredi A, Di Filippo L, ... ... Auricchio A, et al. Therapeutic homology-independent targeted integration in retina and liver. Nature Communications. 13: 1963. PMID 35414130 DOI: 10.1038/s41467-022-29550-8 |
0.366 |
|
| 2021 |
Tornabene P, Trapani I, Centrulo M, Marrocco E, Minopoli R, Lupo M, Iodice C, Gesualdo C, Simonelli F, Surace EM, Auricchio A. Inclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteinsplicing in the retina. Molecular Therapy. Methods & Clinical Development. 23: 448-459. PMID 34786437 DOI: 10.1016/j.omtm.2021.10.004 |
0.395 |
|
| 2021 |
Patrizi C, Llado M, Benati D, Iodice C, Marrocco E, Guarascio R, Surace EM, Cheetham ME, Auricchio A, Recchia A. Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse model. American Journal of Human Genetics. 108: 295-308. PMID 33508235 DOI: 10.1016/j.ajhg.2021.01.006 |
0.348 |
|
| 2020 |
Di Pardo A, Ciaglia E, Cattaneo M, Maciag A, Montella F, Lopardo V, Ferrario A, Villa F, Madonna M, Amico E, Carrizzo A, Damato A, Pepe G, Marracino F, Auricchio A, et al. The longevity-associated variant of BPIFB4 improves a CXCR4-mediated striatum-microglia crosstalk preventing disease progression in a mouse model of Huntington's disease. Cell Death & Disease. 11: 546. PMID 32683420 DOI: 10.1038/S41419-020-02754-W |
0.339 |
|
| 2020 |
Trapani I, Tornabene P, Auricchio A. Large gene delivery to the retina with AAV vectors: are we there yet? Gene Therapy. PMID 32661283 DOI: 10.1038/S41434-020-0174-4 |
0.488 |
|
| 2019 |
Ginocchio VM, Ferla R, Auricchio A, Brunetti-Pierri N. Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism. Human Gene Therapy. PMID 31517544 DOI: 10.1089/Hum.2019.151 |
0.448 |
|
| 2019 |
Puca AA, Carrizzo A, Spinelli C, Damato A, Ambrosio M, Villa F, Ferrario A, Maciag A, Fornai F, Lenzi P, Valenti V, di Nonno F, Accarino G, Madonna M, Forte M, ... ... Auricchio A, et al. Single systemic transfer of a human gene associated with exceptional longevity halts the progression of atherosclerosis and inflammation in ApoE knockout mice through a CXCR4-mediated mechanism. European Heart Journal. PMID 31289820 DOI: 10.1093/Eurheartj/Ehz459 |
0.426 |
|
| 2019 |
Trapani I, Auricchio A. Has retinal gene therapy come of age? From bench to bedside and back to bench. Human Molecular Genetics. PMID 31238338 DOI: 10.1093/Hmg/Ddz130 |
0.497 |
|
| 2019 |
Tornabene P, Trapani I, Minopoli R, Centrulo M, Lupo M, de Simone S, Tiberi P, Dell'Aquila F, Marrocco E, Iodice C, Iuliano A, Gesualdo C, Rossi S, Giaquinto L, Albert S, ... ... Auricchio A, et al. Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina. Science Translational Medicine. 11. PMID 31092694 DOI: 10.1126/Scitranslmed.Aav4523 |
0.523 |
|
| 2018 |
Trapani I, Auricchio A. Seeing the Light after 25 Years of Retinal Gene Therapy. Trends in Molecular Medicine. PMID 29983335 DOI: 10.1016/J.Molmed.2018.06.006 |
0.498 |
|
| 2018 |
Maddalena A, Dell'Aquila F, Giovannelli P, Tiberi P, Wanderlingh L, Montefusco S, Iodice C, Visconte F, Carissimo A, Medina DL, Castoria G, Auricchio A. High-throughput screening identifies kinase inhibitors that increase dual AAV vectors transduction in vitro and in mouse retina. Human Gene Therapy. PMID 29641320 DOI: 10.1089/Hum.2017.220 |
0.371 |
|
| 2017 |
Maddalena A, Tornabene P, Tiberi P, Minopoli R, Manfredi A, Mutarelli M, Rossi S, Simonelli F, Naggert JK, Cacchiarelli D, Auricchio A. Triple Vectors Expand AAV Transfer Capacity in the Retina. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 29292161 DOI: 10.1016/J.Ymthe.2017.11.019 |
0.509 |
|
| 2017 |
Gatto F, Rossi B, Tarallo A, Polishchuk E, Polishchuk R, Carrella A, Nusco E, Alvino FG, Iacobellis F, De Leonibus E, Auricchio A, Diez-Roux G, Ballabio A, Parenti G. AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease. Scientific Reports. 7: 15089. PMID 29118420 DOI: 10.1038/S41598-017-15352-2 |
0.341 |
|
| 2017 |
Ferla R, Alliegro M, Marteau JB, Dell'Anno M, Nusco E, Pouillot S, Galimberti S, Valsecchi MG, Zuliani V, Auricchio A. Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI. Molecular Therapy. Methods & Clinical Development. 6: 143-158. PMID 28932756 DOI: 10.1016/J.Omtm.2017.07.004 |
0.419 |
|
| 2017 |
Ali RR, Auricchio A, Smith AJ. The future looks brighter after 25 years of retinal gene therapy. Human Gene Therapy. PMID 28825330 DOI: 10.1089/Hum.2017.164 |
0.524 |
|
| 2017 |
Bongianino R, Denegri M, Mazzanti A, Lodola F, Vollero A, Boncompagni S, Fasciano S, Rizzo G, Mangione D, Barbaro S, Di Fonso A, Napolitano C, Auricchio A, Protasi F, Priori SG. Allele Specific Silencing of Mutant mRNA Rescues Ultrastructural and Arrhythmic Phenotype in Mice Carriers of the R4496C Mutation in the Ryanodine Receptor Gene (RYR2). Circulation Research. PMID 28620067 DOI: 10.1161/Circresaha.117.310882 |
0.366 |
|
| 2016 |
Testa F, Melillo P, Bonnet C, Marcelli V, de Benedictis A, Colucci R, Gallo B, Kurtenbach A, Rossi S, Marciano E, Auricchio A, Petit C, Zrenner E, Simonelli F. CLINICAL PRESENTATION AND DISEASE COURSE OF USHER SYNDROME BECAUSE OF MUTATIONS IN MYO7A OR USH2A. Retina (Philadelphia, Pa.). PMID 27828912 DOI: 10.1097/Iae.0000000000001389 |
0.303 |
|
| 2016 |
Alliegro M, Ferla R, Nusco E, De Leonibus C, Settembre C, Auricchio A. Low-dose gene therapy reduces the frequency of enzyme replacement therapy in a mouse model of lysosomal storage disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 27658524 DOI: 10.1038/Mt.2016.181 |
0.386 |
|
| 2016 |
Bonnet C, Riahi Z, Chantot-Bastaraud S, Smagghe L, Letexier M, Marcaillou C, Lefèvre GM, Hardelin JP, El-Amraoui A, Singh-Estivalet A, Mohand-Saïd S, Kohl S, Kurtenbach A, Sliesoraityte I, Zobor D, ... ... Auricchio A, et al. An innovative strategy for the molecular diagnosis of Usher syndrome identifies causal biallelic mutations in 93% of European patients. European Journal of Human Genetics : Ejhg. PMID 27460420 DOI: 10.1038/Ejhg.2016.99 |
0.378 |
|
| 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 |
0.433 |
|
| 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 |
0.372 |
|
| 2016 |
Ferla R, Marteau J, Pouillot S, Kozarsky K, Brown J, Galimberti S, Valsecchi MG, Zuliani V, Auricchio A. 468. GLP-Compliant Non-Clinical Safety and Biodistribution of a Recombinant AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI Molecular Therapy. 24: S185. DOI: 10.1016/S1525-0016(16)33277-4 |
0.373 |
|
| 2016 |
Trapani I, Toriello E, Minopoli R, Auricchio A. 187. TFEB-Mediated Clearance of the Lipofuscin Fluorophore A2E Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)32996-3 |
0.332 |
|
| 2016 |
Maddalena A, Tornabene P, Dell'Aquila F, Trapani I, Auricchio A. 28. Mouse and Pig Photoreceptor Transduction Mediated by Triple AAV Vectors Molecular Therapy. 24: S13. DOI: 10.1016/S1525-0016(16)32837-4 |
0.494 |
|
| 2016 |
Casal ML, Wang P, ODonnell PA, Sikora T, Langan T, O'Malley T, Ferla R, Auricchio A, Wilson JM, Gao G, Ponder K, Haskins ME. Urinary glycosaminoglycan concentration as a biomarker for effectiveness of enzyme replacement and gene therapy in large animal models of mucopolysaccharidoses Molecular Genetics and Metabolism. 117: S32. DOI: 10.1016/J.Ymgme.2015.12.214 |
0.478 |
|
| 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 |
0.4 |
|
| 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 |
0.466 |
|
| 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 |
0.519 |
|
| 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 |
0.447 |
|
| 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 |
0.483 |
|
| 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 |
0.308 |
|
| 2015 |
Maddalena A, Colella P, Trapani I, Minopoli R, Auricchio A. 500. Triple AAV Vectors Expand AAV Cargo Capacity in the Retina Molecular Therapy. 23: S200. DOI: 10.1016/S1525-0016(16)34109-0 |
0.491 |
|
| 2015 |
Ferla R, Claudiani P, Savarese M, Kozarsky K, Parini R, Scarpa M, Alice Donati M, Sorge G, Hopwood JJ, Parenti G, Fecarotta S, Nigro V, Serap Sivri H, Van Der Ploeg A, Andria G, ... ... Auricchio A, et al. 372. Prevalence of Anti-AAV8 Neutralizing Antibodies and ARSB Cross-Reactive Immunologic Material in MPS VI Patients Candidates for a Gene Therapy Trial Molecular Therapy. 23: S148. DOI: 10.1016/S1525-0016(16)33981-8 |
0.432 |
|
| 2015 |
Trapani I, de Simone S, Iodice C, Colella P, Rossi S, Auricchio A. 309. Optimization of Dual AAV Vectors for Gene Therapy of Inherited Retinal Diseases Molecular Therapy. 23: S124-S125. DOI: 10.1016/S1525-0016(16)33918-1 |
0.548 |
|
| 2015 |
Toriello E, Colella P, Sommella A, Trapani I, Maddalena A, Auricchio A. 308. Inclusion of Heterologous ITRs in Dual AAV Vectors for Retinal Gene Therapy Molecular Therapy. 23: S124. DOI: 10.1016/S1525-0016(16)33917-X |
0.46 |
|
| 2015 |
Ferla R, Alliegro M, Nusco E, Claudiani P, Auricchio A. 280. Combination of Low-Dose Gene Therapy and Monthly Enzyme Replacement Therapy Improves the Phenotype of a Mouse Model of Lysosomal Storage Disease Molecular Therapy. 23: S112. DOI: 10.1016/S1525-0016(16)33889-8 |
0.408 |
|
| 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 |
0.382 |
|
| 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 |
0.334 |
|
| 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 |
0.55 |
|
| 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 |
0.519 |
|
| 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 |
0.452 |
|
| 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 |
0.452 |
|
| 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 |
0.556 |
|
| 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 |
0.41 |
|
| 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 |
0.313 |
|
| 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 |
0.308 |
|
| 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 |
0.549 |
|
| 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 |
0.358 |
|
| 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 |
0.457 |
|
| 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 |
0.507 |
|
| 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 |
0.54 |
|
| 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 |
0.356 |
|
| 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 |
0.435 |
|
| 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 |
0.417 |
|
| 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 |
0.55 |
|
| 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 |
0.344 |
|
| 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 |
0.386 |
|
| 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 |
0.411 |
|
| 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 |
0.535 |
|
| 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 |
0.357 |
|
| 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 |
0.369 |
|
| 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 |
0.381 |
|
| 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 |
0.433 |
|
| 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 |
0.716 |
|
| 2012 |
Haskins M, Sleeper M, Aguirre G, Walkley SU, Knox V, Vite C, Steet R, Gurda B, Wilson J, Auricchio A, Smith L, Simonaro L, Ponder K. Large Animal Models of Lysosomal Storage Diseases: Lessons on the Limits of Gene/Enzyme Therapy Molecular Genetics and Metabolism. 105: S33-S34. DOI: 10.1016/J.Ymgme.2011.11.071 |
0.504 |
|
| 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 |
0.461 |
|
| 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 |
0.421 |
|
| 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 |
0.435 |
|
| 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 |
0.504 |
|
| 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 |
0.403 |
|
| 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 |
0.514 |
|
| 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 |
0.426 |
|
| 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 |
0.493 |
|
| 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 |
0.414 |
|
| 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 |
0.419 |
|
| 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 |
0.42 |
|
| 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 DOI: 10.2174/156652310793180670 |
0.478 |
|
| 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 |
0.377 |
|
| 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 |
0.511 |
|
| 2010 |
Aloj L, Aurilio M, Rinaldi V, Faella A, Annunziata P, Capalbo A, Cotugno G, Auricchio A. Monitoring AAV mediated somatostatin receptor gene transfer by PET with Ga68-DOTATATE Nuclear Medicine and Biology. 37: 724-725. DOI: 10.1016/J.Nucmedbio.2010.04.040 |
0.31 |
|
| 2010 |
Rotundo IL, Lancioni A, Aurino S, Nigro G, Piluso G, Cacciottolo M, Auricchio A, Nigro V. P3.14 New AAVs for the muscle gene therapy in sarcoglycan deficient animals Neuromuscular Disorders. 20: 645-645. DOI: 10.1016/J.Nmd.2010.07.156 |
0.373 |
|
| 2010 |
Mussolino C, Bacci M, Simonelli F, Auricchio A, Surace E. Immunological, Toxicological and Biodistribution Studies in a Photoreceptor Targeting of Porcine Retina Journal of Biotechnology. 150: 468-468. DOI: 10.1016/J.Jbiotec.2010.09.695 |
0.333 |
|
| 2010 |
Mussolino C, Bacci M, Lavitrano M, Simonelli F, Auricchio A, Surace E. Study of Transduction Efficiencies of Three Vector Serotypes (AAV2/5, 2/7, 2/8) for Photoreceptor Targeting in Porcine Retina Journal of Biotechnology. 150: 467-467. DOI: 10.1016/J.Jbiotec.2010.09.693 |
0.413 |
|
| 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 |
0.466 |
|
| 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 |
0.392 |
|
| 2009 |
Tessitore A, Pirozzi M, Auricchio A. Abnormal autophagy, ubiquitination, inflammation and apoptosis are dependent upon lysosomal storage and are useful biomarkers of mucopolysaccharidosis VI. Pathogenetics. 2: 4. PMID 19531206 DOI: 10.1186/1755-8417-2-4 |
0.311 |
|
| 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 |
0.494 |
|
| 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 |
0.375 |
|
| 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 |
0.492 |
|
| 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 |
0.432 |
|
| 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 |
0.476 |
|
| 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 |
0.547 |
|
| 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 |
0.398 |
|
| 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 |
0.541 |
|
| 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 |
0.483 |
|
| 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 |
0.458 |
|
| 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 |
0.375 |
|
| 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 |
0.726 |
|
| 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 |
0.48 |
|
| 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 |
0.354 |
|
| 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 |
0.457 |
|
| 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 |
0.532 |
|
| 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 |
0.392 |
|
| 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 |
0.399 |
|
| 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 |
0.357 |
|
| 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 |
0.431 |
|
| 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 |
0.412 |
|
| 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/Jci75082 |
0.347 |
|
| 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 |
0.375 |
|
| 2006 |
Allocca M, Vicino UD, Domenici L, Rivera VM, Auricchio A. 885. AAV-Mediated Gene Transfer of Constitutively and Pharmacologically-Activated Glial Cell-Derived Neurotrophic Factor Receptor To Inhibit Retinal Degeneration Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.974 |
0.486 |
|
| 2006 |
Cotugno G, Suace EM, Auricchio A. 868. Novel Strategies To Inhibit Ocular Neovascularization Based on Sonic Hedgehog Pathway Blockade Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.956 |
0.402 |
|
| 2006 |
Tessitore A, Parisi F, Allocca M, Di Vicino U, Denti MA, Domenici L, Bozzoni I, Auricchio A. 712. AAV-Mediated Allele-Specific RNA Interference of a Common Dominant Rhodopsin Mutation Causing Retinitis Pigmentosa Molecular Therapy. 13: S275. DOI: 10.1016/J.Ymthe.2006.08.791 |
0.38 |
|
| 2006 |
Tessitore A, Faella A, Parisi F, Haskins M, Auricchio A. 205. AAV-Mediated Gene Transfer to Muscle and Liver of MPS VI Animal Models Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.230 |
0.466 |
|
| 2006 |
Chadderton N, Palfi A, O'Reilly M, Millington-Ward S, Ader M, Hildinger M, Auricchio A, Tuohy G, Humphries P, Kenna PF, Farrar GJ. 1020. AAV Delivery of Suppression and Replacement Constructs for Rhodopsin-Linked Autosomal Dominant Retinitis Pigmentosa Molecular Therapy. 13: S392-S393. DOI: 10.1016/J.Ymthe.2006.08.1115 |
0.483 |
|
| 2006 |
Vitiello C, Auricchio A, Faraso S, Sorrentino N, Di Napoli D, Castaldo S, Nusco E, Aurino S, Saccone V, Piluso G, Nigro V. T.O. 6 Systemic delta-sarcoglycan gene transfer into cardiomyopathic BIO14.6 hamsters by AAV Neuromuscular Disorders. 16: 724. DOI: 10.1016/J.Nmd.2006.05.253 |
0.311 |
|
| 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 |
0.482 |
|
| 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 |
0.533 |
|
| 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 |
0.461 |
|
| 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 |
0.584 |
|
| 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 |
0.409 |
|
| 2005 |
Surace EM, Tessitore A, Cotugno G, Vitale A, Auricchio A. 669. Sonic Hedgehog (SHH) Is Required for Retinal Angiogenesis and Its Inhibition Prevents Retinal Neovascularization Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.209 |
0.317 |
|
| 2005 |
Surace EM, Domenici L, Cortese K, Venturi C, Cotugno G, Vicino UD, Cellerino A, Marigo V, Tacchetti C, Ballabio A, Auricchio A. 435. Functional and Morphological Rescue of the Type I Ocular Albinism Murine Retina Following AAV-Mediated Gene Transfer Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.437 |
0.454 |
|
| 2005 |
Cardone M, Polito V, D'Azzo A, Muenzer J, Auricchio A, Ballabio A, Cosma MP. 250. Correction of Hunter Syndrome in the Ids-Knock-Out Mouse Model by r-AVV Mediated Gene Therapy Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.253 |
0.43 |
|
| 2005 |
Haskins M, O'Malley T, Wang P, Cullen K, Traas A, Tessitore A, Doria M, Auricchio A. 240. Intramuscular Administration of an AAV Vector Expressing 4-Sulfatase (4S) to Newborn Cats With Mucopolysaccaridosis VI: Sulfatase Activity in Muscle, Serum, and Liver Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.243 |
0.345 |
|
| 2005 |
Allocca M, Rex TS, Domenici L, Bennet J, Rivera VM, Auricchio A. 117. AAV-Mediated Erythropoietin Gene Transfer Protects from Genetic and Light-Induced Retinal Degeneration Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.122 |
0.476 |
|
| 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 |
0.305 |
|
| 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 |
0.484 |
|
| 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 |
0.475 |
|
| 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 |
0.481 |
|
| 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 |
0.476 |
|
| 2004 |
Auricchio A, Cotugno G, Formisano P, Beguinot F, Surace EM, Pollock R. 647. AP20817-Inducible Activation of a Chimeric Insulin Receptor Mimics Insulin Action in Hepatocytes Transduced with AAV Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.590 |
0.301 |
|
| 2004 |
Lebherz C, Maguire AM, Auricchio A, Tang W, Aleman TS, Wei Z, Grant RL, Cideciyan AV, Jacobson SG, Wilson JM, Bennett J. 218. Nonhuman Primate Models for Retinal and Choroidal Neovascularization Using AAV2-Mediated Overexpression of Vascular Endothelial Growth Factor Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.160 |
0.481 |
|
| 2004 |
Surace EM, Auricchio A. Corrigendum to “Adeno-associated viral vectors for retinal gene transfer” [Progress in Retinal and Eye Research 22 (2003) 705–719] Progress in Retinal and Eye Research. 23: 251. DOI: 10.1016/J.Preteyeres.2003.11.001 |
0.441 |
|
| 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 |
0.51 |
|
| 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 |
0.577 |
|
| 2003 |
Auricchio A. Pseudotyped AAV vectors for constitutive and regulated gene expression in the eye. Vision Research. 43: 913-8. PMID 12668060 DOI: 10.1016/S0042-6989(02)00676-4 |
0.477 |
|
| 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 |
0.518 |
|
| 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 |
0.599 |
|
| 2003 |
Angeletti B, Löster J, Auricchio A, Gekeler F, Shinoda K, Ballabio A, Graw J, Marigo V. An in vivo doxycycline-controlled expression system for functional studies of the retina. Investigative Ophthalmology & Visual Science. 44: 755-60. PMID 12556410 DOI: 10.1167/Iovs.02-0340 |
0.428 |
|
| 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 |
0.574 |
|
| 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 |
0.55 |
|
| 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 DOI: 10.1006/Mthe.2002.0660 |
0.553 |
|
| 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 |
0.38 |
|
| 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 DOI: 10.1093/Hmg/10.26.3075 |
0.583 |
|
| 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 |
0.469 |
|
| 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 |
0.524 |
|
| 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 |
0.522 |
|
| 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 |
0.522 |
|
| 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 |
0.494 |
|
| 2000 |
Shifrin AL, Auricchio A, Wilson J, Raper SE. Correction of hyperglycemia by adenoviral vector-mediated insulin gene expression in the murine pancreas Journal of the American College of Surgeons. 191: S31. DOI: 10.1016/S1072-7515(00)00463-4 |
0.459 |
|
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