{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,26]],"date-time":"2026-06-26T02:38:45Z","timestamp":1782441525096,"version":"3.54.5"},"reference-count":129,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T00:00:00Z","timestamp":1722470400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T00:00:00Z","timestamp":1722470400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T00:00:00Z","timestamp":1722470400000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T00:00:00Z","timestamp":1722470400000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T00:00:00Z","timestamp":1722470400000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T00:00:00Z","timestamp":1722470400000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T00:00:00Z","timestamp":1722470400000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Experimental Eye Research"],"published-print":{"date-parts":[[2024,8]]},"DOI":"10.1016\/j.exer.2024.109983","type":"journal-article","created":{"date-parts":[[2024,6,26]],"date-time":"2024-06-26T16:38:15Z","timestamp":1719419895000},"page":"109983","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":14,"special_numbering":"C","title":["Recent advancements and applications of ophthalmic gene therapy strategies: A breakthrough in ocular therapeutics"],"prefix":"10.1016","volume":"245","author":[{"given":"Rashmi","family":"Maurya","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Akash","family":"Vikal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raj Kumar","family":"Narang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Preeti","family":"Patel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8470-6622","authenticated-orcid":false,"given":"Balak Das","family":"Kurmi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib1","article-title":"Update on gene therapy clinical trials for choroideremia and potential experimental therapies","volume":"57","author":"Abbouda","year":"2021","journal-title":"Medicina (Kaunas, Lithuania)"},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib2","first-page":"182","article-title":"Clinical correlates of common corneal neovascular diseases: a literature review","volume":"8","author":"Abdelfattah","year":"2015","journal-title":"Int. J. Ophthalmol."},{"issue":"8","key":"10.1016\/j.exer.2024.109983_bib3","doi-asserted-by":"crossref","DOI":"10.3390\/genes12081241","article-title":"An overview of the genetics of ABCA4 retinopathies, an evolving story","volume":"12","author":"Al-Khuzaei","year":"2021","journal-title":"Genes"},{"key":"10.1016\/j.exer.2024.109983_bib4","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1159\/000324077","article-title":"The genetics of the corneal dystrophies","volume":"48","author":"Aldave","year":"2011","journal-title":"Dev. Ophthalmol."},{"issue":"3","key":"10.1016\/j.exer.2024.109983_bib5","doi-asserted-by":"crossref","first-page":"236","DOI":"10.1038\/ng0397-236","article-title":"A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy","volume":"15","author":"Allikmets","year":"1997","journal-title":"Nat. Genet."},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib6","doi-asserted-by":"crossref","first-page":"104","DOI":"10.1016\/j.spen.2017.05.005","article-title":"Juvenile macular degenerations","volume":"24","author":"Altschwager","year":"2017","journal-title":"Semin. Pediatr. Neurol."},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib7","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.joco.2018.01.006","article-title":"Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation","volume":"30","author":"Ameri","year":"2018","journal-title":"J. Curr.Ophthalmol."},{"key":"10.1016\/j.exer.2024.109983_bib8","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1016\/j.visres.2012.09.012","article-title":"Current mutation discovery approaches in Retinitis Pigmentosa","volume":"75","author":"Anasagasti","year":"2012","journal-title":"Vis. Res."},{"issue":"8","key":"10.1016\/j.exer.2024.109983_bib9","doi-asserted-by":"crossref","DOI":"10.3390\/pharmaceutics14081605","article-title":"Lentiviral vectors for ocular gene therapy","volume":"14","author":"Arsenijevic","year":"2022","journal-title":"Pharmaceutics"},{"issue":"21","key":"10.1016\/j.exer.2024.109983_bib10","doi-asserted-by":"crossref","first-page":"2231","DOI":"10.1056\/NEJMoa0802268","article-title":"Effect of gene therapy on visual function in Leber's congenital amaurosis","volume":"358","author":"Bainbridge","year":"2008","journal-title":"N. Engl. J. Med."},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib11","doi-asserted-by":"crossref","first-page":"365","DOI":"10.1080\/13816810.2021.1904421","article-title":"CRISPR Cas9 based genome editing in inherited retinal dystrophies","volume":"42","author":"Bansal","year":"2021","journal-title":"Ophthalmic Genet."},{"issue":"8","key":"10.1016\/j.exer.2024.109983_bib12","doi-asserted-by":"crossref","DOI":"10.3390\/pharmaceutics12080767","article-title":"Adeno-associated virus mediated gene therapy for corneal diseases","volume":"12","author":"Bastola","year":"2020","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.exer.2024.109983_bib13","doi-asserted-by":"crossref","first-page":"67","DOI":"10.2147\/BTT.S302095","article-title":"The current status of gene therapy for the treatment of cancer","volume":"15","author":"Belete","year":"2021","journal-title":"Biol. Targets & Ther."},{"issue":"120","key":"10.1016\/j.exer.2024.109983_bib14","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.3002865","article-title":"AAV2 gene therapy readministration in three adults with congenital blindness","volume":"4","author":"Bennett","year":"2012","journal-title":"Sci. Transl. Med."},{"issue":"10045","key":"10.1016\/j.exer.2024.109983_bib15","doi-asserted-by":"crossref","first-page":"661","DOI":"10.1016\/S0140-6736(16)30371-3","article-title":"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","volume":"388","author":"Bennett","year":"2016","journal-title":"Lancet (London, England)"},{"key":"10.1016\/j.exer.2024.109983_bib16","doi-asserted-by":"crossref","first-page":"787","DOI":"10.1016\/j.exer.2004.12.005","article-title":"Comparison of HIV-1 and EIAV-based lentiviral vectors in corneal transduction","volume":"80","author":"Beutelspacher","year":"2005","journal-title":"Exp. Eye Res."},{"issue":"11","key":"10.1016\/j.exer.2024.109983_bib17","doi-asserted-by":"crossref","first-page":"1224","DOI":"10.1016\/j.addr.2006.07.023","article-title":"Non-viral ocular gene therapy: potential ocular therapeutic avenues","volume":"58","author":"Bloquel","year":"2006","journal-title":"Adv. Drug Deliv. Rev."},{"issue":"8","key":"10.1016\/j.exer.2024.109983_bib18","first-page":"1685","article-title":"Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives","volume":"24","author":"Bordet","year":"2019"},{"issue":"6","key":"10.1016\/j.exer.2024.109983_bib19","doi-asserted-by":"crossref","first-page":"e177","DOI":"10.1016\/j.jfo.2017.05.003","article-title":"Corneal dystrophies","volume":"40","author":"Bourges","year":"2017","journal-title":"J. Fr. Ophtalmol."},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib20","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1080\/2576117X.2020.1760072","article-title":"Congenital nystagmus and its congeners","volume":"70","author":"Brodsky","year":"2020","journal-title":"J. Binocul. Vis. Ocul. Motil."},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib21","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1038\/gt.2011.164","article-title":"Gene transfer for ocular neovascularization and macular edema","volume":"19","author":"Campochiaro","year":"2012","journal-title":"Gene Ther."},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib22","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1089\/hum.2006.17.167","article-title":"Adenoviral vector-delivered pigment epithelium-derived factor for neovascular age-related macular degeneration: results of a phase I clinical trial","volume":"17","author":"Campochiaro","year":"2006","journal-title":"Hum. Gene Ther."},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib23","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1089\/hum.2016.117","article-title":"Lentiviral vector gene transfer of endostatin\/angiostatin for macular degeneration (GEM) study","volume":"28","author":"Campochiaro","year":"2017","journal-title":"Hum. Gene Ther."},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib24","first-page":"73","article-title":"AAV-based strategies for treatment of retinal and choroidal vascular diseases: advances in age-related macular degeneration and diabetic retinopathy therapies","volume":"38","author":"Castro","year":"2024","journal-title":"BioDrugs Clin. Immunother. Biopharmaceut. Gene Ther."},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib25","doi-asserted-by":"crossref","first-page":"267","DOI":"10.2174\/138920211795860116","article-title":"Diagnostic challenges in retinitis pigmentosa: genotypic multiplicity and phenotypic variability","volume":"12","author":"Chang","year":"2011","journal-title":"Curr. Genom."},{"issue":"6","key":"10.1016\/j.exer.2024.109983_bib26","first-page":"587","article-title":"Leber congenital amaurosis: clinical correlations with genotypes, gene therapy trials update, and future directions","volume":"13","author":"Chung","year":"2009","journal-title":"J. AAPOS Offic. Pub. Am. Assoc. Pediatric Ophthalmol. Strabismus."},{"issue":"5","key":"10.1016\/j.exer.2024.109983_bib27","doi-asserted-by":"crossref","first-page":"525","DOI":"10.1093\/hmg\/ddh048","article-title":"Mutations in ABCA4 result in accumulation of lipofuscin before slowing of the retinoid cycle: a reappraisal of the human disease sequence","volume":"13","author":"Cideciyan","year":"2004","journal-title":"Hum. Mol. Genet."},{"issue":"12","key":"10.1016\/j.exer.2024.109983_bib28","doi-asserted-by":"crossref","first-page":"1753","DOI":"10.1016\/j.ophtha.2017.05.035","article-title":"Prevalence of age-related macular degeneration in europe: the past and the future","volume":"124","author":"Colijn","year":"2017","journal-title":"Ophthalmology"},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib29","doi-asserted-by":"crossref","first-page":"104","DOI":"10.22336\/rjo.2021.22","article-title":"Corneal dystrophies: pathophysiological, genetic, clinical, and therapeutic considerations","volume":"65","author":"Constantin","year":"2021","journal-title":"Rom. J.Ophthalmol."},{"issue":"10","key":"10.1016\/j.exer.2024.109983_bib30","doi-asserted-by":"crossref","first-page":"1354","DOI":"10.1001\/archophthalmol.2012.2008","article-title":"Fundus autofluorescence patterns in stargardt disease over time-reply","volume":"130","author":"Cukras","year":"2012","journal-title":"Arch. Ophthalmol."},{"issue":"6","key":"10.1016\/j.exer.2024.109983_bib31","doi-asserted-by":"crossref","first-page":"2177","DOI":"10.1172\/JCI120429","article-title":"Gene therapy and genome surgery in the retina","volume":"128","author":"DiCarlo","year":"2018","journal-title":"J. Clin. Invest."},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib32","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1167\/iovs.12-10050","article-title":"A1120, a nonretinoid RBP4 antagonist, inhibits formation of cytotoxic bisretinoids in the animal model of enhanced retinal lipofuscinogenesis","volume":"54","author":"Dobri","year":"2013","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"issue":"3","key":"10.1016\/j.exer.2024.109983_bib33","first-page":"162","article-title":"Decorin\u2010PEI nanoconstruct attenuates equine corneal fibroblast differentiation","volume":"17","author":"Donnelly","year":"2014"},{"issue":"19","key":"10.1016\/j.exer.2024.109983_bib34","first-page":"1302","article-title":"Mutations within the rhodopsin gene in patients with autosomal dominant retinitis pigmentosa","volume":"323","author":"Dryja","year":"1990"},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib35","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1007\/s00005-010-0063-4","article-title":"Lentiviral vectors in gene therapy: their current status and future potential","volume":"58","author":"Escors","year":"2010","journal-title":"Arch. Immunol. Ther. Exp."},{"key":"10.1016\/j.exer.2024.109983_bib36","doi-asserted-by":"crossref","first-page":"191","DOI":"10.1007\/128_2010_70","article-title":"Cationic liposome-nucleic acid complexes for gene delivery and silencing: pathways and mechanisms for plasmid DNA and siRNA","volume":"296","author":"Ewert","year":"2010","journal-title":"Top. Curr. Chem."},{"issue":"16","key":"10.1016\/j.exer.2024.109983_bib37","doi-asserted-by":"crossref","DOI":"10.3390\/molecules27165089","article-title":"Recent advances in age-related macular degeneration therapies","volume":"27","author":"Fabre","year":"2022","journal-title":"Molecules"},{"issue":"6","key":"10.1016\/j.exer.2024.109983_bib38","doi-asserted-by":"crossref","first-page":"725","DOI":"10.1097\/MOP.0000000000000690","article-title":"Retinitis pigmentosa: recent advances and future directions in diagnosis and management","volume":"30","author":"Fahim","year":"2018","journal-title":"Curr. Opin. Pediatr."},{"issue":"6","key":"10.1016\/j.exer.2024.109983_bib39","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1001\/jamaophthalmol.2020.1032","article-title":"Safety and vision outcomes of subretinal gene therapy targeting cone photoreceptors in achromatopsia: a nonrandomized controlled trial","volume":"138","author":"Fischer","year":"2020","journal-title":"JAMA Ophthalmol."},{"issue":"12","key":"10.1016\/j.exer.2024.109983_bib40","article-title":"Differentially expressed wound healing-related microRNAs in the human diabetic cornea","volume":"8","author":"Funari","year":"2013"},{"issue":"8","key":"10.1016\/j.exer.2024.109983_bib41","doi-asserted-by":"crossref","first-page":"927","DOI":"10.1097\/ICO.0b013e318201405a","article-title":"Treatments for corneal neovascularization: a review","volume":"30","author":"Gupta","year":"2011","journal-title":"Cornea"},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib42","doi-asserted-by":"crossref","first-page":"414","DOI":"10.1002\/jnr.490340406","article-title":"A developmentally regulated microsomal protein specific for the pigment epithelium of the vertebrate retina","volume":"34","author":"Hamel","year":"1993","journal-title":"J. Neurosci. Res."},{"issue":"10","key":"10.1016\/j.exer.2024.109983_bib43","doi-asserted-by":"crossref","first-page":"979","DOI":"10.1089\/hum.2008.107","article-title":"Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial","volume":"19","author":"Hauswirth","year":"2008","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.exer.2024.109983_bib44","doi-asserted-by":"crossref","first-page":"291","DOI":"10.34133\/research.0291","article-title":"AAV for gene therapy in ocular diseases: progress and prospects","volume":"6","author":"He","year":"2023","journal-title":"Research"},{"key":"10.1016\/j.exer.2024.109983_bib45","series-title":"MCO-010 Optogenetic Gene Therapy for Severe Vision Loss in Stargardt Disease","author":"Ho","year":"2023"},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib46","first-page":"8279","article-title":"Molecular diagnosis of 34 Japanese families with leber congenital amaurosis using targeted next generation sequencing","volume":"8","author":"Hosono","year":"2018"},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib47","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1080\/13816810.2021.1966053","article-title":"Stargardt disease and progress in therapeutic strategies","volume":"43","author":"Huang","year":"2022","journal-title":"Ophthalmic Genet."},{"issue":"5","key":"10.1016\/j.exer.2024.109983_bib48","doi-asserted-by":"crossref","first-page":"839","DOI":"10.1016\/j.neuron.2019.02.017","article-title":"Therapeutic AAV gene transfer to the nervous system: a clinical reality","volume":"101","author":"Hudry","year":"2019","journal-title":"Neuron"},{"issue":"3","key":"10.1016\/j.exer.2024.109983_bib49","doi-asserted-by":"crossref","first-page":"1382","DOI":"10.1167\/iovs.09-3860","article-title":"Human corneal fibrosis: an in vitro model","volume":"51","author":"Karamichos","year":"2010","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib50","doi-asserted-by":"crossref","first-page":"241","DOI":"10.2174\/156652311796150363","article-title":"Advances and future challenges in adenoviral vector pharmacology and targeting","volume":"11","author":"Khare","year":"2011","journal-title":"Curr. Gene Ther."},{"issue":"7","key":"10.1016\/j.exer.2024.109983_bib51","doi-asserted-by":"crossref","first-page":"1814","DOI":"10.1016\/j.biomaterials.2009.10.031","article-title":"Ultrapure chitosan oligomers as carriers for corneal gene transfer","volume":"31","author":"Klausner","year":"2010","journal-title":"Biomaterials"},{"issue":"10","key":"10.1016\/j.exer.2024.109983_bib52","first-page":"429","article-title":"Current and emerging therapies for ocular herpes simplex virus type-1 infections","volume":"7","author":"Koganti","year":"2019"},{"issue":"7603","key":"10.1016\/j.exer.2024.109983_bib53","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib54","first-page":"341","article-title":"[Vectors used in gene therapy]","volume":"99","author":"Kremen","year":"1998","journal-title":"Sb. Lek."},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib55","doi-asserted-by":"crossref","first-page":"335","DOI":"10.1016\/j.ophtha.2014.08.032","article-title":"Early-onset stargardt disease: phenotypic and genotypic characteristics","volume":"122","author":"Lambertus","year":"2015","journal-title":"Ophthalmology"},{"issue":"13","key":"10.1016\/j.exer.2024.109983_bib56","doi-asserted-by":"crossref","first-page":"5186","DOI":"10.1167\/iovs.16-19833","article-title":"Progression of late-onset stargardt disease","volume":"57","author":"Lambertus","year":"2016","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib57","doi-asserted-by":"crossref","first-page":"292","DOI":"10.1038\/sj.gt.3302861","article-title":"Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium","volume":"14","author":"Le","year":"2007","journal-title":"Gene Ther."},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib58","doi-asserted-by":"crossref","first-page":"500","DOI":"10.1001\/archopht.123.4.500","article-title":"Postsurgical assessment and long-term safety of recombinant adeno-associated virus-mediated gene transfer into the retinas of dogs and primates","volume":"123","author":"Le Meur","year":"2005","journal-title":"Arch. Ophthalmol."},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib59","first-page":"43","article-title":"Adenovirus-mediated gene delivery: potential applications for gene and cell-based therapies in the new era of personalized medicine","volume":"4","author":"Lee","year":"2017","journal-title":"Gene. amp; Dis."},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib60","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1038\/s41551-019-0501-5","article-title":"Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses","volume":"4","author":"Levy","year":"2020","journal-title":"Nat. Biomed. Eng."},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib61","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a017384","article-title":"Leber congenital amaurosis caused by mutations in RPGRIP1","volume":"5","author":"Li","year":"2014","journal-title":"Cold Spring Harb.Perspect. Med."},{"issue":"5567","key":"10.1016\/j.exer.2024.109983_bib62","first-page":"497","article-title":"Murine leukemia induced by retroviral gene marking","volume":"296","author":"Li","year":"2002"},{"issue":"3","key":"10.1016\/j.exer.2024.109983_bib63","first-page":"1336","article-title":"Mutation survey of known LCA genes and loci in the Saudi Arabian population","volume":"50","author":"Li","year":"2009"},{"issue":"10","key":"10.1016\/j.exer.2024.109983_bib64","doi-asserted-by":"crossref","first-page":"2120","DOI":"10.1016\/j.ymthe.2020.06.029","article-title":"Gene therapy intervention in neovascular eye disease: a recent update","volume":"28","author":"Lin","year":"2020","journal-title":"Mol. Ther. : J.Am.Soc.Gene Ther."},{"key":"10.1016\/j.exer.2024.109983_bib65","doi-asserted-by":"crossref","DOI":"10.1016\/j.exer.2019.107715","article-title":"Clinical and genetic update of corneal dystrophies","volume":"186","author":"Lisch","year":"2019","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.exer.2024.109983_bib66","doi-asserted-by":"crossref","DOI":"10.1016\/j.exer.2020.108139","article-title":"Early and late clinical landmarks of corneal dystrophies","volume":"198","author":"Lisch","year":"2020","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.exer.2024.109983_bib67","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1016\/j.preteyeres.2015.07.002","article-title":"Progress in corneal wound healing","volume":"49","author":"Ljubimov","year":"2015","journal-title":"Prog. Retin. Eye Res."},{"issue":"6","key":"10.1016\/j.exer.2024.109983_bib68","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a017319","article-title":"Gene therapy for the retinal degeneration of usher syndrome caused by mutations in MYO7A","volume":"5","author":"Lopes","year":"2015","journal-title":"Cold Spring Harb.Perspect. Med."},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib69","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1038\/s41591-018-0327-9","article-title":"Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10","volume":"25","author":"Maeder","year":"2019","journal-title":"Nat. Med."},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib70","first-page":"229","article-title":"Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10","volume":"25","author":"Maeder","year":"2019"},{"issue":"21","key":"10.1016\/j.exer.2024.109983_bib71","doi-asserted-by":"crossref","first-page":"2240","DOI":"10.1056\/NEJMoa0802315","article-title":"Safety and efficacy of gene transfer for Leber's congenital amaurosis","volume":"358","author":"Maguire","year":"2008","journal-title":"N. Engl. J. Med."},{"issue":"9701","key":"10.1016\/j.exer.2024.109983_bib72","doi-asserted-by":"crossref","first-page":"1597","DOI":"10.1016\/S0140-6736(09)61836-5","article-title":"Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial","volume":"374","author":"Maguire","year":"2009","journal-title":"Lancet (London, England)"},{"issue":"9","key":"10.1016\/j.exer.2024.109983_bib73","first-page":"1273","article-title":"Efficacy, safety, and durability of voretigene neparvovec-rzyl in RPE65 mutation\u2013associated inherited retinal dystrophy: results of phase 1 and 3 trials","volume":"126","author":"Maguire","year":"2019"},{"key":"10.1016\/j.exer.2024.109983_bib74","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1016\/j.omtm.2020.03.002","article-title":"Germline CRISPR\/Cas9-Mediated gene editing prevents vision loss in a novel mouse model of aniridia","volume":"17","author":"Mirjalili Mohanna","year":"2020","journal-title":"Mol. Ther.Methods & Clin. Dev."},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib75","first-page":"238","article-title":"Decorin transfection suppresses profibrogenic genes and myofibroblast formation in human corneal fibroblasts","volume":"91","author":"Mohan","year":"2010"},{"key":"10.1016\/j.exer.2024.109983_bib76","doi-asserted-by":"crossref","DOI":"10.1016\/j.exer.2020.108361","article-title":"Novel insights into gene therapy in the cornea","volume":"202","author":"Mohan","year":"2021","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.exer.2024.109983_bib77","first-page":"478","article-title":"Germline CRISPR\/Cas9-mediated gene editing prevents vision loss in a novel mouse model of aniridia","volume":"17","author":"Mohanna","year":"2020"},{"issue":"10","key":"10.1016\/j.exer.2024.109983_bib78","doi-asserted-by":"crossref","first-page":"1235","DOI":"10.1080\/14712598.2017.1356817","article-title":"Gene therapy for age-related macular degeneration","volume":"17","author":"Moore","year":"2017","journal-title":"Expet Opin. Biol. Ther."},{"key":"10.1016\/j.exer.2024.109983_bib79","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1016\/j.preteyeres.2018.01.004","article-title":"Personalised genome editing - the future for corneal dystrophies","volume":"65","author":"Moore","year":"2018","journal-title":"Prog. Retin. Eye Res."},{"issue":"10","key":"10.1016\/j.exer.2024.109983_bib80","doi-asserted-by":"crossref","DOI":"10.3390\/v12101106","article-title":"Gene therapy applications of non-human lentiviral vectors","volume":"12","author":"Munis","year":"2020","journal-title":"Viruses"},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib81","doi-asserted-by":"crossref","first-page":"317","DOI":"10.1007\/s40259-017-0234-5","article-title":"Adeno-associated virus (AAV) as a vector for gene therapy","volume":"31","author":"Naso","year":"2017","journal-title":"BioDrugs : Clin.Immunother. biopharmaceuticals Gene Ther."},{"key":"10.1016\/j.exer.2024.109983_bib82","doi-asserted-by":"crossref","first-page":"27","DOI":"10.4103\/2277-9175.98152","article-title":"Viral and nonviral delivery systems for gene delivery","volume":"1","author":"Nayerossadat","year":"2012","journal-title":"Adv. Biomed. Res."},{"issue":"Suppl. 1","key":"10.1016\/j.exer.2024.109983_bib83","doi-asserted-by":"crossref","first-page":"S18","DOI":"10.4103\/0974-620X.122290","article-title":"Hereditary retinal eye diseases in childhood and youth affecting the central retina","volume":"6","author":"Nentwich","year":"2013","journal-title":"Oman J. Ophthalmol."},{"issue":"15","key":"10.1016\/j.exer.2024.109983_bib84","doi-asserted-by":"crossref","first-page":"1278","DOI":"10.21037\/atm-20-4726","article-title":"Gene therapy for inherited retinal diseases","volume":"9","author":"Nuzbrokh","year":"2021","journal-title":"Ann. Transl. Med."},{"key":"10.1016\/j.exer.2024.109983_bib85","series-title":"Polysaccharide Carriers for Drug Delivery","first-page":"79","article-title":"4 - chitosan-based nanocarriers for ophthalmic applications","author":"Paliwal","year":"2019"},{"key":"10.1016\/j.exer.2024.109983_bib86","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1016\/j.ajo.2022.02.013","article-title":"Three-year safety results of SAR422459 (EIAV-ABCA4) gene therapy in patients with ABCA4-associated stargardt disease: an open-label dose-escalation phase I\/IIa clinical trial, cohorts 1-5","volume":"240","author":"Parker","year":"2022","journal-title":"Am. J. Ophthalmol."},{"issue":"21","key":"10.1016\/j.exer.2024.109983_bib87","doi-asserted-by":"crossref","DOI":"10.3390\/ijms20215491","article-title":"The development of functional non-viral vectors for gene delivery","volume":"20","author":"Patil","year":"2019","journal-title":"Int. J. Mol. Sci."},{"issue":"121","key":"10.1016\/j.exer.2024.109983_bib88","first-page":"221","article-title":"Gene therapy approaches for the treatment of retinal disorders","volume":"22","author":"Petit","year":"2016","journal-title":"Discov. Med."},{"issue":"8","key":"10.1016\/j.exer.2024.109983_bib89","doi-asserted-by":"crossref","first-page":"563","DOI":"10.1089\/hum.2016.040","article-title":"Advances in gene therapy for diseases of the eye","volume":"27","author":"Petit","year":"2016","journal-title":"Hum. Gene Ther."},{"issue":"3","key":"10.1016\/j.exer.2024.109983_bib90","first-page":"147","article-title":"Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease","volume":"31","author":"Prado","year":"2020"},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib91","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1021\/acsomega.2c05976","article-title":"Surface modification of lipid-based nanocarriers: a potential approach to enhance targeted drug delivery","volume":"8","author":"Priya","year":"2023","journal-title":"ACS Omega"},{"issue":"16","key":"10.1016\/j.exer.2024.109983_bib92","doi-asserted-by":"crossref","DOI":"10.3390\/polym14163410","article-title":"Chitosan-hyaluronic acid nanoparticles for active targeting in cancer therapy","volume":"14","author":"Puluhulawa","year":"2022","journal-title":"Polymers"},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib93","first-page":"Ge01","article-title":"Non viral vectors in gene therapy- an overview","volume":"9","author":"Ramamoorth","year":"2015","journal-title":"J. Clin. Diagn. Res. : J. Clin. Diagn. Res."},{"issue":"283","key":"10.1016\/j.exer.2024.109983_bib94","first-page":"19","article-title":"Leading causes of blindness worldwide","author":"Roodhooft","year":"2002","journal-title":"Bull. Soc. Belge Ophtalmol."},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib95","doi-asserted-by":"crossref","first-page":"398","DOI":"10.1016\/j.jtos.2018.06.004","article-title":"Current and emerging therapies for corneal neovascularization","volume":"16","author":"Roshandel","year":"2018","journal-title":"Ocul. Surf."},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib96","doi-asserted-by":"crossref","first-page":"157","DOI":"10.18240\/ijo.2022.01.23","article-title":"Progress of clinical therapies for dry age-related macular degeneration","volume":"15","author":"Rubner","year":"2022","journal-title":"Int. J. Ophthalmol."},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib97","doi-asserted-by":"crossref","first-page":"1970","DOI":"10.1167\/iovs.09-4569","article-title":"Normalization of wound healing and diabetic markers in organ cultured human diabetic corneas by adenoviral delivery of c-Met gene","volume":"51","author":"Saghizadeh","year":"2010","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"issue":"9","key":"10.1016\/j.exer.2024.109983_bib98","first-page":"1280","article-title":"Corneal regeneration using gene therapy approaches","volume":"12","author":"Sarkar","year":"2023"},{"key":"10.1016\/j.exer.2024.109983_bib99","article-title":"The yin and yang of the opioid growth regulatory system: focus on diabetes\u2014the lorenz E","volume":"2016","author":"Sassani","year":"2016","journal-title":"Zimmerman Tribute Lect."},{"issue":"5","key":"10.1016\/j.exer.2024.109983_bib100","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1167\/tvst.6.5.6","article-title":"Towards treatment of stargardt disease: workshop organized and sponsored by the foundation fighting blindness","volume":"6","author":"Sears","year":"2017","journal-title":"Transl. Vis. Sci. &Technol."},{"key":"10.1016\/j.exer.2024.109983_bib101","first-page":"22","volume":"vol 181","author":"Serratrice","year":"2014"},{"issue":"5","key":"10.1016\/j.exer.2024.109983_bib102","doi-asserted-by":"crossref","first-page":"891","DOI":"10.4161\/rna.23764","article-title":"Protospacer recognition motifs: mixed identities and functional diversity","volume":"10","author":"Shah","year":"2013","journal-title":"RNA Biol."},{"issue":"5","key":"10.1016\/j.exer.2024.109983_bib103","doi-asserted-by":"crossref","DOI":"10.3390\/biom13050724","article-title":"The autoimmune rheumatic disease related dry eye and its association with retinopathy","volume":"13","author":"Shan","year":"2023","journal-title":"Biomolecules"},{"issue":"3","key":"10.1016\/j.exer.2024.109983_bib104","first-page":"480","article-title":"Leber's congenital amaurosis and the role of gene therapy in congenital retinal disorders","volume":"10","author":"Sharif","year":"2017","journal-title":"Int. J. Ophthalmol."},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib105","doi-asserted-by":"crossref","first-page":"15","DOI":"10.22336\/rjo.2019.4","article-title":"Corneal neovascularization: updates on pathophysiology, investigations & management","volume":"63","author":"Sharif","year":"2019","journal-title":"Rom. j.Ophthalmol."},{"key":"10.1016\/j.exer.2024.109983_bib106","series-title":"Encyclopedia of the Eye","first-page":"185","article-title":"Gene therapy for the cornea, conjunctiva, and lacrimal gland","author":"Sharma","year":"2010"},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib107","doi-asserted-by":"crossref","first-page":"505","DOI":"10.1016\/j.nano.2011.01.006","article-title":"Polyethylenimine-conjugated gold nanoparticles: gene transfer potential and low toxicity in the cornea","volume":"7","author":"Sharma","year":"2011","journal-title":"Nanomed. Nanotechnol. Biol. Med."},{"key":"10.1016\/j.exer.2024.109983_bib108","article-title":"A mini-review on new developments in nanocarriers and polymers for ophthalmic drug delivery strategies","author":"Sharma","year":"2023","journal-title":"Curr. Drug Deliv."},{"issue":"9","key":"10.1016\/j.exer.2024.109983_bib109","doi-asserted-by":"crossref","first-page":"4284","DOI":"10.1167\/iovs.07-0068","article-title":"Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients","volume":"48","author":"Simonelli","year":"2007","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"issue":"3","key":"10.1016\/j.exer.2024.109983_bib110","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1038\/mt.2009.277","article-title":"Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration","volume":"18","author":"Simonelli","year":"2010","journal-title":"Mol. Ther. : J.Am.Soc.Gene Ther."},{"key":"10.1016\/j.exer.2024.109983_bib111","first-page":"15","volume":"vol 178","author":"Simunovic","year":"2019"},{"issue":"Pt B","key":"10.1016\/j.exer.2024.109983_bib112","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1016\/j.ejpb.2014.12.022","article-title":"Treatment of ocular disorders by gene therapy","volume":"95","author":"Solin\u00eds","year":"2015","journal-title":"Eur. J. Pharm. Biopharm."},{"issue":"29\u201330","key":"10.1016\/j.exer.2024.109983_bib113","first-page":"513","article-title":"The diagnosis and treatment of age-related macular degeneration","volume":"117","author":"Stahl","year":"2020","journal-title":"Deut. Arzteblatt Int."},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib114","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1136\/bjophthalmol-2016-308823","article-title":"Stargardt disease: clinical features, molecular genetics, animal models and therapeutic options","volume":"101","author":"Tanna","year":"2017","journal-title":"Br. J. Ophthalmol."},{"issue":"6","key":"10.1016\/j.exer.2024.109983_bib115","first-page":"652","article-title":"The genetic profile of Leber congenital amaurosis in an Australian cohort","volume":"5","author":"Thompson","year":"2017"},{"issue":"7","key":"10.1016\/j.exer.2024.109983_bib116","doi-asserted-by":"crossref","first-page":"2280","DOI":"10.4103\/ijo.IJO_2255_21","article-title":"Clinical and molecular aspects of congenital aniridia - a review of current concepts","volume":"70","author":"Tibrewal","year":"2022","journal-title":"Indian J. Ophthalmol."},{"key":"10.1016\/j.exer.2024.109983_bib117","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1186\/1740-2557-2-6","article-title":"Expression of TNF inhibitor gene in the lacrimal gland promotes recovery of tear production and tear stability and reduced immunopathology in rabbits with induced autoimmune dacryoadenitis","volume":"2","author":"Trousdale","year":"2005","journal-title":"J. Autoimmune Dis."},{"key":"10.1016\/j.exer.2024.109983_bib118","doi-asserted-by":"crossref","first-page":"147","DOI":"10.3329\/bsmmuj.v7i2.29452","article-title":"Case series of Stargardt's disease: our experience","volume":"7","author":"Wadud","year":"2016","journal-title":"Bangabandhu Sheikh Mujib Med. Univ. J."},{"issue":"6","key":"10.1016\/j.exer.2024.109983_bib119","first-page":"3642","article-title":"Comprehensive molecular diagnosis of a large Chinese Leber congenital amaurosis cohort","volume":"56","author":"Wang","year":"2015"},{"issue":"2","key":"10.1016\/j.exer.2024.109983_bib120","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1167\/tvst.8.2.21","article-title":"An ophthalmic targeted exome sequencing panel as a powerful tool to identify causative mutations in patients suspected of hereditary eye diseases","volume":"8","author":"Wang","year":"2019","journal-title":"Transl. Vis. Sci. &Technol."},{"issue":"4","key":"10.1016\/j.exer.2024.109983_bib121","doi-asserted-by":"crossref","first-page":"276","DOI":"10.1097\/ICU.0b013e3283541eb6","article-title":"Gene therapy for corneal dystrophies and disease, where are we?","volume":"23","author":"Williams","year":"2012","journal-title":"Curr. Opin. Ophthalmol."},{"issue":"12","key":"10.1016\/j.exer.2024.109983_bib122","article-title":"Targeting miR-146a to treat delayed wound healing in human diabetic organ-cultured corneas","volume":"9","author":"Winkler","year":"2014"},{"issue":"3","key":"10.1016\/j.exer.2024.109983_bib123","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1111\/j.1753-4887.2005.tb00127.x","article-title":"Function of the protein RPE65 in the visual cycle","volume":"63","author":"Wolf","year":"2005","journal-title":"Nutr. Rev."},{"issue":"10","key":"10.1016\/j.exer.2024.109983_bib124","doi-asserted-by":"crossref","first-page":"1322","DOI":"10.1136\/bjophthalmol-2015-308329","article-title":"Genetic manipulation for inherited neurodegenerative diseases: myth or reality?","volume":"100","author":"Yu-Wai-Man","year":"2016","journal-title":"Br. J. Ophthalmol."},{"issue":"9","key":"10.1016\/j.exer.2024.109983_bib125","doi-asserted-by":"crossref","first-page":"709","DOI":"10.1136\/jmg.40.9.709","article-title":"A Tyr368His <em>RPE65<\/em> founder mutation is associated with variable expression and progression of early onset retinal dystrophy in 10 families of a genetically isolated population","volume":"40","author":"Yzer","year":"2003","journal-title":"J. Med. Genet."},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib126","doi-asserted-by":"crossref","DOI":"10.1080\/20022727.2018.1488497","article-title":"Polyethylenimine-based nanocarriers in co-delivery of drug and gene: a developing horizon","volume":"9","author":"Zakeri","year":"2018","journal-title":"Nano Rev. & Exp."},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib127","doi-asserted-by":"crossref","first-page":"1","DOI":"10.5582\/irdr.2020.01011","article-title":"A basic understanding of mucopolysaccharidosis: incidence, clinical features, diagnosis, and management","volume":"9","author":"Zhou","year":"2020","journal-title":"Intractable & rare diseases research"},{"issue":"1","key":"10.1016\/j.exer.2024.109983_bib128","first-page":"33","article-title":"Kinetics of keratocyte proliferation in response to epithelial debridement","volume":"72","author":"Zieske","year":"2001"},{"key":"10.1016\/j.exer.2024.109983_bib129","volume":"vol 6","author":"Zwerling","year":"2021"}],"container-title":["Experimental Eye Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014483524002045?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014483524002045?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,8,23]],"date-time":"2024-08-23T00:54:08Z","timestamp":1724374448000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0014483524002045"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,8]]},"references-count":129,"alternative-id":["S0014483524002045"],"URL":"https:\/\/doi.org\/10.1016\/j.exer.2024.109983","relation":{},"ISSN":["0014-4835"],"issn-type":[{"value":"0014-4835","type":"print"}],"subject":[],"published":{"date-parts":[[2024,8]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Recent advancements and applications of ophthalmic gene therapy strategies: A breakthrough in ocular therapeutics","name":"articletitle","label":"Article Title"},{"value":"Experimental Eye Research","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.exer.2024.109983","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 Elsevier Ltd. All rights are reserved, including those for text and data mining, AI training, and similar technologies.","name":"copyright","label":"Copyright"}],"article-number":"109983"}}