{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T16:54:51Z","timestamp":1791996891707,"version":"4.4.0"},"reference-count":34,"publisher":"Association for Research in Vision and Ophthalmology (ARVO)","issue":"15","license":[{"start":{"date-parts":[[2023,12,22]],"date-time":"2023-12-22T00:00:00Z","timestamp":1703203200000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Invest. Ophthalmol. Vis. Sci."],"published-print":{"date-parts":[[2023,12,22]]},"DOI":"10.1167\/iovs.64.15.31","type":"journal-article","created":{"date-parts":[[2023,12,22]],"date-time":"2023-12-22T09:31:02Z","timestamp":1703237462000},"page":"31","source":"Crossref","is-referenced-by-count":13,"title":["The Use of rAAV2-RB1\u2013Mediated Gene Therapy in Retinoblastoma"],"prefix":"10.1167","volume":"64","author":[{"given":"Hanhan","family":"Shi","sequence":"first","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoyu","family":"He","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhi","family":"Yang","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qili","family":"Liao","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jing","family":"Ruan","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shengfang","family":"Ge","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peiwei","family":"Chai","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renbing","family":"Jia","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jiayan","family":"Fan","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xuyang","family":"Wen","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xianqun","family":"Fan","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai, People's Republic of China"},{"name":"Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology, Shanghai, People's Republic of China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"27","reference":[{"key":"bib1","doi-asserted-by":"crossref","first-page":"685","DOI":"10.1001\/jamaoncol.2019.6716","article-title":"Global retinoblastoma presentation and analysis by national income level","volume":"6","author":"Global Retinoblastoma Study G","year":"2020","journal-title":"JAMA Oncol"},{"key":"bib2","doi-asserted-by":"publisher","first-page":"341","DOI":"10.1002\/pbc.v56.3","article-title":"Strategies to manage retinoblastoma in developing countries","volume":"56","author":"Chantada","year":"2011","journal-title":"Pediatr Blood Cancer"},{"key":"bib3","doi-asserted-by":"publisher","first-page":"1341","DOI":"10.1001\/jamaophthalmol.2015.3108","article-title":"Treatment of retinoblastoma in 2015: agreement and disagreement","volume":"133","author":"Abramson","year":"2015","journal-title":"JAMA Ophthalmol"},{"key":"bib4","doi-asserted-by":"publisher","first-page":"1455","DOI":"10.1001\/jamaophthalmol.2015.2861","article-title":"Anterior segment seeding in eyes with retinoblastoma failing to respond to intraophthalmic artery chemotherapy","volume":"133","author":"Pavlidou","year":"2015","journal-title":"JAMA Ophthalmol"},{"key":"bib5","doi-asserted-by":"publisher","first-page":"875","DOI":"10.1016\/j.ajo.2014.07.025","article-title":"Retinoblastoma. Fifty years of progress. The LXXI Edward Jackson Memorial Lecture","volume":"158","author":"Grossniklaus","year":"2014","journal-title":"Am J Ophthalmol"},{"key":"bib6","doi-asserted-by":"publisher","first-page":"329","DOI":"10.1038\/nature10733","article-title":"A novel retinoblastoma therapy from genomic and epigenetic analyses","volume":"481","author":"Zhang","year":"2012","journal-title":"Nature"},{"key":"bib7","doi-asserted-by":"publisher","first-page":"15021","DOI":"10.1038\/nrdp.2015.21","article-title":"Retinoblastoma","volume":"1","author":"Dimaras","year":"2015","journal-title":"Nat Rev Dis Primers"},{"key":"bib8","doi-asserted-by":"publisher","first-page":"501","DOI":"10.1126\/science.3983638","article-title":"Genetic origin of mutations predisposing to retinoblastoma","volume":"228","author":"Cavenee","year":"1985","journal-title":"Science"},{"key":"bib9","doi-asserted-by":"publisher","first-page":"643","DOI":"10.1038\/323643a0","article-title":"A human DNA segment with properties of the gene that predisposes to retinoblastoma and osteosarcoma","volume":"323","author":"Friend","year":"1986","journal-title":"Nature"},{"key":"bib10","doi-asserted-by":"publisher","first-page":"385","DOI":"10.1038\/nature13813","article-title":"Rb suppresses human cone-precursor-derived retinoblastoma tumours","volume":"514","author":"Xu","year":"2014","journal-title":"Nature"},{"key":"bib11","doi-asserted-by":"publisher","first-page":"438","DOI":"10.18632\/oncotarget.v5i2","article-title":"RB1 gene inactivation by chromothripsis in human retinoblastoma","volume":"5","author":"McEvoy","year":"2014","journal-title":"Oncotarget"},{"key":"bib12","doi-asserted-by":"publisher","first-page":"820","DOI":"10.1073\/pnas.68.4.820","article-title":"Mutation and cancer: statistical study of retinoblastoma","volume":"68","author":"Knudson","year":"1971","journal-title":"Proc Natl Acad Sci USA"},{"key":"bib13","doi-asserted-by":"publisher","first-page":"488","DOI":"10.1016\/j.ophtha.2016.12.015","article-title":"Efficacy and toxicity of intravitreous chemotherapy for retinoblastoma: four-year experience","volume":"124","author":"Francis","year":"2017","journal-title":"Ophthalmology"},{"key":"bib14","doi-asserted-by":"publisher","first-page":"209","DOI":"10.1111\/aos.2014.92.issue-3","article-title":"Intra-arterial chemotherapy for retinoblastoma. Challenges of a prospective study","volume":"92","author":"Parareda","year":"2014","journal-title":"Acta Ophthalmol"},{"key":"bib15","doi-asserted-by":"publisher","first-page":"1223","DOI":"10.1007\/s00247-016-3554-6","article-title":"A review of the literature for intra-arterial chemotherapy used to treat retinoblastoma","volume":"46","author":"Wyse","year":"2016","journal-title":"Pediatr Radiol"},{"key":"bib16","doi-asserted-by":"publisher","first-page":"351","DOI":"10.1038\/ng1318","article-title":"Rb regulates proliferation and rod photoreceptor development in the mouse retina","volume":"36","author":"Zhang","year":"2004","journal-title":"Nat Genet"},{"key":"bib17","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1038\/ng0501-92","article-title":"Gene therapy restores vision in a canine model of childhood blindness","volume":"28","author":"Acland","year":"2001","journal-title":"Nat Genet"},{"key":"bib18","doi-asserted-by":"publisher","first-page":"98","DOI":"10.1353\/pbm.1970.0011","article-title":"The use of DNA for gene therapy\u2013the need, experimental approach, and implications","volume":"14","author":"Aposhian","year":"1970","journal-title":"Perspect Biol Med"},{"key":"bib19","doi-asserted-by":"publisher","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"key":"bib20","doi-asserted-by":"publisher","first-page":"215870","DOI":"10.1016\/j.canlet.2022.215870","article-title":"Novel insights into RB1 mutation","volume":"547","author":"Yao","year":"2022","journal-title":"Cancer Lett"},{"key":"bib21","doi-asserted-by":"publisher","first-page":"235","DOI":"10.2174\/1566523217666171003170348","article-title":"Comparative study of adeno-associated virus, adenovirus, bacu lovirus and lentivirus vectors for gene therapy of the eyes","volume":"17","author":"Kalesnykas","year":"2017","journal-title":"Curr Gene Ther"},{"key":"bib22","doi-asserted-by":"publisher","first-page":"671","DOI":"10.1080\/13816810.2018.1533027","article-title":"Gene therapy for RPE65-related retinal disease","volume":"39","author":"Miraldi Utz","year":"2018","journal-title":"Ophthalmic Genet"},{"key":"bib23","doi-asserted-by":"publisher","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","article-title":"Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial","volume":"390","author":"Russell","year":"2017","journal-title":"Lancet"},{"key":"bib24","doi-asserted-by":"publisher","first-page":"464","DOI":"10.1016\/j.ymthe.2020.12.007","article-title":"Current clinical applications of in vivo gene therapy with AAVs","volume":"29","author":"Mendell","year":"2021","journal-title":"Mol Ther"},{"key":"bib25","doi-asserted-by":"publisher","first-page":"173","DOI":"10.1038\/d41573-021-00017-7","article-title":"The clinical landscape for AAV gene therapies","volume":"20","author":"Kuzmin","year":"2021","journal-title":"Nat Rev Drug Discov"},{"key":"bib26","doi-asserted-by":"publisher","first-page":"1558","DOI":"10.1016\/j.ophtha.2006.03.039","article-title":"Metastatic retinoblastoma clinical features, treatment, and prognosis","volume":"113","author":"Gunduz","year":"2006","journal-title":"Ophthalmology"},{"issue":"Suppl 1","key":"bib27","doi-asserted-by":"crossref","first-page":"S10","DOI":"10.1038\/sj.gt.3302364","article-title":"Immune responses to gene therapy vectors: influence on vector function and effector mechanisms","volume":"11","author":"Bessis","year":"2004","journal-title":"Gene Ther"},{"key":"bib28","doi-asserted-by":"publisher","first-page":"eabm8466","DOI":"10.1126\/sciadv.abm8466","article-title":"RB1 loss triggers dependence on ESRRG in retinoblastoma","volume":"8","author":"Field","year":"2022","journal-title":"Sci Adv"},{"key":"bib29","doi-asserted-by":"publisher","first-page":"1051","DOI":"10.1038\/4661051a","article-title":"Cell cycle: retinoblastoma, a trip organizer","volume":"466","author":"Bosco","year":"2010","journal-title":"Nature"},{"key":"bib30","doi-asserted-by":"publisher","first-page":"eaat9321","DOI":"10.1126\/scitranslmed.aat9321","article-title":"Therapeutic targeting of the RB1 pathway in retinoblastoma with the oncolytic adenovirus VCN-01","volume":"11:","author":"Pascual-Pasto","year":"2019","journal-title":"Sci Transl Med"},{"key":"bib31","doi-asserted-by":"publisher","first-page":"5","DOI":"10.1016\/j.cell.2018.03.021","article-title":"Gene therapy for retinal degeneration","volume":"173","author":"Apte","year":"2018","journal-title":"Cell"},{"key":"bib32","doi-asserted-by":"publisher","first-page":"10","DOI":"10.1016\/j.preteyeres.2012.10.002","article-title":"Immunological homeostasis of the eye","volume":"33","author":"Mochizuki","year":"2013","journal-title":"Prog Retin Eye Res"},{"key":"bib33","doi-asserted-by":"publisher","first-page":"e135112","DOI":"10.1172\/jci.insight.135112","article-title":"In vivo-directed evolution of adeno-associated virus in the primate retina","volume":"5","author":"Byrne","year":"2020","journal-title":"JCI Insight"},{"key":"bib34","doi-asserted-by":"publisher","first-page":"189ra176","DOI":"10.1126\/scitranslmed.3005708","article-title":"In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous","volume":"5","author":"Dalkara","year":"2013","journal-title":"Sci Transl Med"}],"container-title":["Investigative Opthalmology &amp; Visual Science"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/iovs.arvojournals.org\/article.aspx?articleid=2793217","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T21:31:42Z","timestamp":1791927102000},"score":1,"resource":{"primary":{"URL":"https:\/\/iovs.arvojournals.org\/article.aspx?articleid=2793217"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,12,22]]},"references-count":34,"journal-issue":{"issue":"15","published-print":{"date-parts":[[2023,12,1]]}},"URL":"https:\/\/doi.org\/10.1167\/iovs.64.15.31","relation":{},"ISSN":["1552-5783"],"issn-type":[{"value":"1552-5783","type":"print"}],"subject":[],"published":{"date-parts":[[2023,12,22]]}}}