{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T19:16:09Z","timestamp":1793301369151,"version":"4.5.0"},"reference-count":85,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,5,26]],"date-time":"2026-05-26T00:00:00Z","timestamp":1779753600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100031367","name":"Choroideremia Research Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100031367","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100014069","name":"Champalimaud Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100014069","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001871","name":"Funda\u00e7\u00e3o para a Ci\u00eancia e a Tecnologia","doi-asserted-by":"publisher","award":["2022.12254.BD"],"award-info":[{"award-number":["2022.12254.BD"]}],"id":[{"id":"10.13039\/501100001871","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001871","name":"Funda\u00e7\u00e3o para a Ci\u00eancia e a Tecnologia","doi-asserted-by":"publisher","award":["UID\/04443\/2025"],"award-info":[{"award-number":["UID\/04443\/2025"]}],"id":[{"id":"10.13039\/501100001871","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Advances"],"published-print":{"date-parts":[[2026,9]]},"DOI":"10.1016\/j.omta.2026.201768","type":"journal-article","created":{"date-parts":[[2026,5,28]],"date-time":"2026-05-28T15:12:45Z","timestamp":1779981165000},"page":"201768","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":2,"title":["mRNA delivery to the retina restores REP1 function in choroideremia"],"prefix":"10.1016","volume":"34","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4666-5388","authenticated-orcid":false,"given":"Luisa","family":"de Lemos","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pedro","family":"Antas","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cl\u00e1udia","family":"Carvalho","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariana","family":"Castro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diogo","family":"Bruno","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana F.","family":"Fonseca","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shuvajit","family":"Rakshit","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6404-4892","authenticated-orcid":false,"given":"Miguel C.","family":"Seabra","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omta.2026.201768_bib1","doi-asserted-by":"crossref","first-page":"335","DOI":"10.1016\/j.preteyeres.2010.03.004","article-title":"The molecular basis of human retinal and vitreoretinal diseases","volume":"29","author":"Berger","year":"2010","journal-title":"Prog. Retin. Eye Res."},{"key":"10.1016\/j.omta.2026.201768_bib2","doi-asserted-by":"crossref","DOI":"10.1016\/j.preteyeres.2021.101029","article-title":"Inherited retinal diseases: Linking genes, disease-causing variants, and relevant therapeutic modalities","volume":"89","author":"Schneider","year":"2022","journal-title":"Prog. Retin. Eye Res."},{"key":"10.1016\/j.omta.2026.201768_bib3","first-page":"52","article-title":"Emerging Treatments for Retinitis Pigmentosa: Genes and stem cells, as well as new electronic and medical therapies, are gaining ground","volume":"12","author":"Lin","year":"2015","journal-title":"Retin. Physician"},{"key":"10.1016\/j.omta.2026.201768_bib4","doi-asserted-by":"crossref","first-page":"113","DOI":"10.3109\/13816810.2013.841962","article-title":"Bilateral Concordance of the Fundus Hyperautofluorescent Ring in Typical Retinitis Pigmentosa Patients","volume":"36","author":"Sujirakul","year":"2015","journal-title":"Ophthalmic Genet."},{"key":"10.1016\/j.omta.2026.201768_bib5","doi-asserted-by":"crossref","first-page":"296","DOI":"10.3389\/fimmu.2012.00296","article-title":"The privileged immunity of immune privileged organs: the case of the eye","volume":"3","author":"Benhar","year":"2012","journal-title":"Front. Immunol."},{"key":"10.1016\/j.omta.2026.201768_bib6","doi-asserted-by":"crossref","first-page":"444","DOI":"10.1080\/08164622.2021.1880863","article-title":"Gene therapy for inherited retinal diseases: progress and possibilities","volume":"104","author":"Hu","year":"2021","journal-title":"Clin. Exp. Optom."},{"key":"10.1016\/j.omta.2026.201768_bib7","doi-asserted-by":"crossref","first-page":"1706","DOI":"10.3390\/cells13201706","article-title":"A Comparative Analysis of Models for AAV-Mediated Gene Therapy for Inherited Retinal Diseases","volume":"13","author":"Alsalloum","year":"2024","journal-title":"Cells"},{"key":"10.1016\/j.omta.2026.201768_bib8","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2025.1664968","article-title":"Immune responses in retinal gene therapy: challenges, mechanisms, and future strategies","volume":"16","author":"Ma","year":"2025","journal-title":"Front. Immunol."},{"key":"10.1016\/j.omta.2026.201768_bib9","doi-asserted-by":"crossref","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 Lond. Engl."},{"key":"10.1016\/j.omta.2026.201768_bib10","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\u2019s congenital amaurosis: a phase 1 dose-escalation trial","volume":"374","author":"Maguire","year":"2009","journal-title":"Lancet Lond. Engl."},{"key":"10.1016\/j.omta.2026.201768_bib11","doi-asserted-by":"crossref","first-page":"1283","DOI":"10.1016\/j.ophtha.2012.11.048","article-title":"Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2","volume":"120","author":"Testa","year":"2013","journal-title":"Ophthalmology"},{"key":"10.1016\/j.omta.2026.201768_bib12","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1007\/s11095-018-2564-5","article-title":"Adeno-Associated Viral Gene Therapy for Inherited Retinal Disease","volume":"36","author":"Ong","year":"2019","journal-title":"Pharm. Res."},{"key":"10.1016\/j.omta.2026.201768_bib13","doi-asserted-by":"crossref","first-page":"2240","DOI":"10.1056\/NEJMoa0802315","article-title":"Safety and efficacy of gene transfer for Leber\u2019s congenital amaurosis","volume":"358","author":"Maguire","year":"2008","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omta.2026.201768_bib14","doi-asserted-by":"crossref","first-page":"1287","DOI":"10.1016\/j.ophtha.2022.06.018","article-title":"Inflammation after Voretigene Neparvovec Administration in Patients with RPE65-Related Retinal Dystrophy","volume":"129","author":"Kessel","year":"2022","journal-title":"Ophthalmology"},{"key":"10.1016\/j.omta.2026.201768_bib15","doi-asserted-by":"crossref","first-page":"1331","DOI":"10.1136\/bjophthalmol-2021-321023","article-title":"Development of retinal atrophy after subretinal gene therapy with voretigene neparvovec","volume":"107","author":"Reichel","year":"2023","journal-title":"Br. J. Ophthalmol."},{"key":"10.1016\/j.omta.2026.201768_bib16","doi-asserted-by":"crossref","first-page":"58","DOI":"10.1016\/j.oret.2021.03.016","article-title":"Perifoveal Chorioretinal Atrophy after Subretinal Voretigene Neparvovec-rzyl for RPE65-Mediated Leber Congenital Amaurosis","volume":"6","author":"Gange","year":"2022","journal-title":"Ophthalmol. Retina"},{"key":"10.1016\/j.omta.2026.201768_bib17","doi-asserted-by":"crossref","first-page":"1543","DOI":"10.1007\/s00417-021-05508-2","article-title":"Real-world outcomes of voretigene neparvovec treatment in pediatric patients with RPE65-associated Leber congenital amaurosis","volume":"260","author":"Deng","year":"2022","journal-title":"Graefes Arch. Clin. Exp. Ophthalmol."},{"key":"10.1016\/j.omta.2026.201768_bib18","doi-asserted-by":"crossref","first-page":"1920","DOI":"10.1056\/NEJMoa1412965","article-title":"Improvement and decline in vision with gene therapy in childhood blindness","volume":"372","author":"Jacobson","year":"2015","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omta.2026.201768_bib19","doi-asserted-by":"crossref","first-page":"1887","DOI":"10.1056\/NEJMoa1414221","article-title":"Long-term effect of gene therapy on Leber\u2019s congenital amaurosis","volume":"372","author":"Bainbridge","year":"2015","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omta.2026.201768_bib20","doi-asserted-by":"crossref","first-page":"1104","DOI":"10.1038\/ng.3407","article-title":"Adeno-associated virus finds its disease","volume":"47","author":"Russell","year":"2015","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omta.2026.201768_bib21","doi-asserted-by":"crossref","first-page":"472","DOI":"10.1038\/s41591-025-04073-z","article-title":"Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy","volume":"32","author":"Buddle","year":"2026","journal-title":"Nat. Med."},{"key":"10.1016\/j.omta.2026.201768_bib22","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1016\/j.ejpb.2014.12.028","article-title":"Nanoparticle-based technologies for retinal gene therapy","volume":"95","author":"Adijanto","year":"2015","journal-title":"Eur. J. Pharm. Biopharm."},{"key":"10.1016\/j.omta.2026.201768_bib23","doi-asserted-by":"crossref","first-page":"1361","DOI":"10.2147\/IJN.S157000","article-title":"DNA nanoparticles are safe and nontoxic in non-human primate eyes","volume":"13","author":"Kelley","year":"2018","journal-title":"Int. J. Nanomedicine"},{"key":"10.1016\/j.omta.2026.201768_bib24","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1016\/j.ijpharm.2010.01.019","article-title":"Lipid-mediated delivery of RNA is more efficient than delivery of DNA in non-dividing cells","volume":"389","author":"Zou","year":"2010","journal-title":"Int. J. Pharm. X."},{"key":"10.1016\/j.omta.2026.201768_bib25","doi-asserted-by":"crossref","first-page":"166","DOI":"10.1038\/s41392-022-01007-w","article-title":"mRNA-based therapeutics: powerful and versatile tools to combat diseases","volume":"7","author":"Qin","year":"2022","journal-title":"Signal Transduct. Targeted Ther."},{"key":"10.1016\/j.omta.2026.201768_bib26","doi-asserted-by":"crossref","DOI":"10.34133\/bmr.0178","article-title":"Current Trends in Messenger RNA Technology for Cancer Therapeutics","volume":"29","author":"Afzal","year":"2025","journal-title":"Biomater. Res."},{"key":"10.1016\/j.omta.2026.201768_bib27","doi-asserted-by":"crossref","DOI":"10.1016\/j.addr.2023.115026","article-title":"Gene editing therapeutics based on mRNA delivery","volume":"200","author":"Popovitz","year":"2023","journal-title":"Adv. Drug Deliv. Rev."},{"key":"10.1016\/j.omta.2026.201768_bib28","doi-asserted-by":"crossref","first-page":"817","DOI":"10.1038\/s41573-021-00283-5","article-title":"mRNA vaccines for infectious diseases: principles, delivery and clinical translation","volume":"20","author":"Chaudhary","year":"2021","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omta.2026.201768_bib29","doi-asserted-by":"crossref","first-page":"785","DOI":"10.1016\/j.ymthe.2018.11.018","article-title":"mRNA-Based Protein Replacement Therapy for the Heart","volume":"27","author":"Magadum","year":"2019","journal-title":"Mol. Ther. J. Am. Soc. Gene Ther."},{"key":"10.1016\/j.omta.2026.201768_bib30","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1016\/j.jconrel.2019.06.042","article-title":"Non-viral delivery of chemically modified mRNA to the retina: Subretinal versus intravitreal administration","volume":"307","author":"Devoldere","year":"2019","journal-title":"J. Contr. Release"},{"key":"10.1016\/j.omta.2026.201768_bib31","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.add4623","article-title":"Peptide-guided lipid nanoparticles deliver mRNA to the neural retina of rodents and nonhuman primates","volume":"9","author":"Herrera-Barrera","year":"2023","journal-title":"Sci. Adv."},{"key":"10.1016\/j.omta.2026.201768_bib32","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1016\/j.jconrel.2019.04.015","article-title":"Lipid nanoparticles for delivery of messenger RNA to the back of the eye","volume":"303","author":"Patel","year":"2019","journal-title":"J. Contr. Release"},{"key":"10.1016\/j.omta.2026.201768_bib33","first-page":"622","article-title":"Deletions in patients with classical choroideremia vary in size from 45 kb to several megabases","volume":"47","author":"Cremers","year":"1990","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.omta.2026.201768_bib34","doi-asserted-by":"crossref","first-page":"43","DOI":"10.3109\/13816819609057869","article-title":"New insights into the pathogenesis of choroideremia: a tale of two REPs","volume":"17","author":"Seabra","year":"1996","journal-title":"Ophthalmic Genet."},{"key":"10.1016\/j.omta.2026.201768_bib35","doi-asserted-by":"crossref","first-page":"1547","DOI":"10.1167\/iovs.18-25501","article-title":"CHM\/REP1 Transcript Expression and Loss of Visual Function in Patients Affected by Choroideremia","volume":"60","author":"Di Iorio","year":"2019","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.omta.2026.201768_bib36","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1167\/tvst.10.8.12","article-title":"Expression of Rab Prenylation Pathway Genes and Relation to Disease Progression in Choroideremia","volume":"10","author":"Fry","year":"2021","journal-title":"Transl. Vis. Sci. Technol."},{"key":"10.1016\/j.omta.2026.201768_bib37","series-title":"Gene Therapy for Choroideremia Using an Adeno-Associated Viral Vector Encoding Rab Escort Protein 1: The REGENERATE Open-Label Trial","author":"Cehajic-Kapetanovic","year":"2024"},{"key":"10.1016\/j.omta.2026.201768_bib38","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1038\/s41587-025-02556-5","article-title":"Safer non-viral DNA delivery using lipid nanoparticles loaded with endogenous anti-inflammatory lipids","volume":"44","author":"Patel","year":"2025","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omta.2026.201768_bib39","doi-asserted-by":"crossref","first-page":"759","DOI":"10.1038\/nrd4278","article-title":"mRNA-based therapeutics--developing a new class of drugs","volume":"13","author":"Sahin","year":"2014","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omta.2026.201768_bib40","doi-asserted-by":"crossref","first-page":"24420","DOI":"10.1074\/jbc.270.41.24420","article-title":"Deficient geranylgeranylation of Ram\/Rab27 in choroideremia","volume":"270","author":"Seabra","year":"1995","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.omta.2026.201768_bib41","doi-asserted-by":"crossref","first-page":"760","DOI":"10.3390\/biom11050760","article-title":"A Systematic Review and Meta-Analyses of Interventional Clinical Trial Studies for Gene Therapies for the Inherited Retinal Degenerations (IRDs)","volume":"11","author":"Tuohy","year":"2021","journal-title":"Biomolecules"},{"key":"10.1016\/j.omta.2026.201768_bib42","doi-asserted-by":"crossref","first-page":"487","DOI":"10.1007\/s40291-020-00475-6","article-title":"Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy","volume":"24","author":"Kang","year":"2020","journal-title":"Mol. Diagn. Ther."},{"key":"10.1016\/j.omta.2026.201768_bib43","doi-asserted-by":"crossref","first-page":"2464","DOI":"10.1038\/s41591-023-02520-3","article-title":"Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial","volume":"29","author":"MacLaren","year":"2023","journal-title":"Nat. Med."},{"key":"10.1016\/j.omta.2026.201768_bib44","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1038\/nn.2467","article-title":"A robust and high-throughput Cre reporting and characterization system for the whole mouse brain","volume":"13","author":"Madisen","year":"2010","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.omta.2026.201768_bib45","doi-asserted-by":"crossref","first-page":"4717","DOI":"10.3390\/jcm11164717","article-title":"Subretinal Injection Techniques for Retinal Disease: A Review","volume":"11","author":"Irigoyen","year":"2022","journal-title":"J. Clin. Med."},{"key":"10.1016\/j.omta.2026.201768_bib46","doi-asserted-by":"crossref","DOI":"10.3389\/fvets.2025.1574519","article-title":"Advances in technical methods and applications of subretinal injections in experimental animals","volume":"12","author":"Song","year":"2025","journal-title":"Front. Vet. Sci."},{"key":"10.1016\/j.omta.2026.201768_bib47","article-title":"mRNA-lipid nanoparticle COVID-19 vaccines: Structure and stability","volume":"601","author":"Schoenmaker","year":"2021","journal-title":"Int. J. Pharm. X."},{"key":"10.1016\/j.omta.2026.201768_bib48","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1002\/(SICI)1520-6017(200004)89:4<443::AID-JPS2>3.0.CO;2-W","article-title":"Chemical stability of nucleic acid-derived drugs","volume":"89","author":"Pogocki","year":"2000","journal-title":"J. Pharm. Sci."},{"key":"10.1016\/j.omta.2026.201768_bib49","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1167\/tvst.13.7.7","article-title":"Lipid nanoparticle-mediated delivery of mRNA into the mouse and human retina and other ocular tissues","volume":"13","author":"Chambers","year":"2024","journal-title":"Transl. Vis. Sci. Technol."},{"key":"10.1016\/j.omta.2026.201768_bib50","doi-asserted-by":"crossref","first-page":"366","DOI":"10.1111\/ceo.13702","article-title":"Local delivery of corticosteroids in clinical ophthalmology: A review","volume":"48","author":"Fung","year":"2020","journal-title":"Clin. Exp. Ophthalmol."},{"key":"10.1016\/j.omta.2026.201768_bib51","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1097\/IIO.0000000000000366","article-title":"Ocular Inflammation and Treatment Emergent Adverse Events in Retinal Gene Therapy","volume":"61","author":"Mehta","year":"2021","journal-title":"Int. Ophthalmol. Clin."},{"key":"10.1016\/j.omta.2026.201768_bib52","doi-asserted-by":"crossref","first-page":"5785","DOI":"10.1073\/pnas.1821000116","article-title":"AAV cis-regulatory sequences are correlated with ocular toxicity","volume":"116","author":"Xiong","year":"2019","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omta.2026.201768_bib53","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0242284","article-title":"Chronically shortened rod outer segments accompany photoreceptor cell death in Choroideremia","volume":"15","author":"Meschede","year":"2020","journal-title":"PLoS One"},{"key":"10.1016\/j.omta.2026.201768_bib54","doi-asserted-by":"crossref","first-page":"386","DOI":"10.1172\/JCI26617","article-title":"Independent degeneration of photoreceptors and retinal pigment epithelium in conditional knockout mouse models of choroideremia","volume":"116","author":"Tolmachova","year":"2006","journal-title":"J. Clin. Investig."},{"key":"10.1016\/j.omta.2026.201768_bib55","doi-asserted-by":"crossref","DOI":"10.3389\/fopht.2023.1270561","article-title":"Gene therapy for inherited retinal diseases: exploiting new tools in genome editing and nanotechnology","volume":"3","author":"Carvalho","year":"2023","journal-title":"Front. Ophthalmol."},{"key":"10.1016\/j.omta.2026.201768_bib56","doi-asserted-by":"crossref","first-page":"136","DOI":"10.1016\/j.molmed.2023.11.009","article-title":"Toward low-cost gene therapy: mRNA-based therapeutics for treatment of inherited retinal diseases","volume":"30","author":"Antas","year":"2024","journal-title":"Trends Mol. Med."},{"key":"10.1016\/j.omta.2026.201768_bib57","doi-asserted-by":"crossref","first-page":"1049","DOI":"10.1016\/0092-8674(92)90253-9","article-title":"Purification of component A of Rab geranylgeranyl transferase: possible identity with the choroideremia gene product","volume":"70","author":"Seabra","year":"1992","journal-title":"Cell"},{"key":"10.1016\/j.omta.2026.201768_bib58","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1126\/science.8380507","article-title":"Retinal degeneration in choroideremia: deficiency of rab geranylgeranyl transferase","volume":"259","author":"Seabra","year":"1993","journal-title":"Science"},{"key":"10.1016\/j.omta.2026.201768_bib59","doi-asserted-by":"crossref","first-page":"1556","DOI":"10.3390\/jcm9051556","article-title":"Preoperative, Intraoperative and Postoperative Corticosteroid Use as an Adjunctive Treatment for Rhegmatogenous Retinal Detachment","volume":"9","author":"Bonfiglio","year":"2020","journal-title":"J. Clin. Med."},{"key":"10.1016\/j.omta.2026.201768_bib60","doi-asserted-by":"crossref","first-page":"1461","DOI":"10.1080\/10717544.2020.1833382","article-title":"A sustained dual drug delivery system for proliferative vitreoretinopathy","volume":"27","author":"Xiao","year":"2020","journal-title":"Drug Deliv."},{"key":"10.1016\/j.omta.2026.201768_bib61","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1016\/j.jconrel.2015.08.051","article-title":"N(1)-methylpseudouridine-incorporated mRNA outperforms pseudouridine-incorporated mRNA by providing enhanced protein expression and reduced immunogenicity in mammalian cell lines and mice","volume":"217","author":"Andries","year":"2015","journal-title":"J. Contr. Release"},{"key":"10.1016\/j.omta.2026.201768_bib62","doi-asserted-by":"crossref","first-page":"1101","DOI":"10.1002\/jbm.a.37356","article-title":"Rational design of anti-inflammatory lipid nanoparticles for mRNA delivery","volume":"110","author":"Zhang","year":"2022","journal-title":"J. Biomed. Mater. Res."},{"key":"10.1016\/j.omta.2026.201768_bib63","article-title":"The Pivotal Role of Chemical Modifications in mRNA Therapeutics","volume":"10","author":"Liu","year":"2022","journal-title":"Front. Cell Dev. Biol."},{"key":"10.1016\/j.omta.2026.201768_bib64","doi-asserted-by":"crossref","first-page":"530","DOI":"10.1089\/hum.2022.222","article-title":"The Degree of Adeno-Associated Virus-Induced Retinal Inflammation Varies Based on Serotype and Route of Delivery: Intravitreal, Subretinal, or Suprachoroidal. Hum","volume":"34","author":"Wiley","year":"2023","journal-title":"Gene Ther."},{"key":"10.1016\/j.omta.2026.201768_bib65","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.adv7922","article-title":"Retinal gene therapy using epiretinal AAV-containing fibrin hydrogel implants","volume":"11","author":"Scruggs","year":"2025","journal-title":"Sci. Adv."},{"key":"10.1016\/j.omta.2026.201768_bib66","doi-asserted-by":"crossref","first-page":"1574","DOI":"10.1038\/sj.gt.3300994","article-title":"Immune responses to adenovirus and adeno-associated virus in humans","volume":"6","author":"Chirmule","year":"1999","journal-title":"Gene Ther."},{"key":"10.1016\/j.omta.2026.201768_bib67","doi-asserted-by":"crossref","first-page":"1197","DOI":"10.3390\/pharmaceutics16091197","article-title":"Advances in Nanoparticles as Non-Viral Vectors for Efficient Delivery of CRISPR\/Cas9","volume":"16","author":"Kim","year":"2024","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.omta.2026.201768_bib68","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1186\/s12951-025-03095-6","article-title":"Frontier applications of retinal nanomedicine: progress, challenges and perspectives","volume":"23","author":"Tang","year":"2025","journal-title":"J. Nanobiotechnol."},{"key":"10.1016\/j.omta.2026.201768_bib69","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1016\/j.jconrel.2022.07.002","article-title":"ICG-mediated photodisruption of the inner limiting membrane enhances retinal drug delivery","volume":"349","author":"Peynshaert","year":"2022","journal-title":"J. Control. Release"},{"key":"10.1016\/j.omta.2026.201768_bib70","doi-asserted-by":"crossref","first-page":"1716","DOI":"10.3390\/pharmaceutics14081716","article-title":"Photodisruption of the Inner Limiting Membrane: Exploring ICG Loaded Nanoparticles as Photosensitizers","volume":"14","author":"De Clerck","year":"2022","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.omta.2026.201768_bib71","doi-asserted-by":"crossref","first-page":"5746","DOI":"10.1039\/C9CS00839J","article-title":"Stimuli-responsive nanobubbles for biomedical applications","volume":"50","author":"Xiong","year":"2021","journal-title":"Chem. Soc. Rev."},{"key":"10.1016\/j.omta.2026.201768_bib72","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0008402","article-title":"Loss-of-function mutations in Rab escort protein 1 (REP-1) affect intracellular transport in fibroblasts and monocytes of choroideremia patients","volume":"4","author":"Strunnikova","year":"2009","journal-title":"PLoS One"},{"key":"10.1016\/j.omta.2026.201768_bib73","doi-asserted-by":"crossref","DOI":"10.1073\/pnas.2307796121","article-title":"Targeted nonviral delivery of genome editors in vivo","volume":"121","author":"Tsuchida","year":"2024","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omta.2026.201768_bib74","doi-asserted-by":"crossref","first-page":"1059","DOI":"10.1002\/anie.201610209","article-title":"Non-Viral CRISPR\/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA","volume":"56","author":"Miller","year":"2017","journal-title":"Angew. Chem. Int. Ed."},{"key":"10.1016\/j.omta.2026.201768_bib75","doi-asserted-by":"crossref","first-page":"318","DOI":"10.3390\/vaccines12030318","article-title":"Self-Amplifying RNA: A Second Revolution of mRNA Vaccines against COVID-19","volume":"12","author":"Silva-Pilipich","year":"2024","journal-title":"Vaccines"},{"key":"10.1016\/j.omta.2026.201768_bib76","doi-asserted-by":"crossref","first-page":"566","DOI":"10.3390\/v17040566","article-title":"Self-Amplifying RNA: Advantages and Challenges of a Versatile Platform for Vaccine Development","volume":"17","author":"Vallet","year":"2025","journal-title":"Viruses"},{"key":"10.1016\/j.omta.2026.201768_bib77","doi-asserted-by":"crossref","first-page":"516","DOI":"10.1159\/000529207","article-title":"Circular RNA in Retina: A Potential Biomarker and Therapeutic Target","volume":"66","author":"Tian","year":"2023","journal-title":"Ophthalmic Res."},{"key":"10.1016\/j.omta.2026.201768_bib78","doi-asserted-by":"crossref","first-page":"2629","DOI":"10.1038\/s41467-018-05096-6","article-title":"Engineering circular RNA for potent and stable translation in eukaryotic cells","volume":"9","author":"Wesselhoeft","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omta.2026.201768_bib79","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1023\/A:1008868325009","article-title":"Maternally expressed PGK-Cre transgene as a tool for early and uniform activation of the Cre site-specific recombinase","volume":"7","author":"Lallemand","year":"1998","journal-title":"Transgenic Res."},{"key":"10.1016\/j.omta.2026.201768_bib80","doi-asserted-by":"crossref","first-page":"5635","DOI":"10.1016\/j.cell.2021.09.018","article-title":"Enhanced prime editing systems by manipulating cellular determinants of editing outcomes","volume":"184","author":"Chen","year":"2021","journal-title":"Cell"},{"key":"10.1016\/j.omta.2026.201768_bib81","doi-asserted-by":"crossref","first-page":"2431","DOI":"10.1038\/s41596-022-00724-4","article-title":"Designing and executing prime editing experiments in mammalian cells","volume":"17","author":"Doman","year":"2022","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.omta.2026.201768_bib82","doi-asserted-by":"crossref","first-page":"528","DOI":"10.1089\/scd.2024.0105","article-title":"Modeling Choroideremia Disease with Isogenic Induced Pluripotent Stem Cells","volume":"33","author":"Fonseca","year":"2024","journal-title":"Stem Cell. Dev."},{"key":"10.1016\/j.omta.2026.201768_bib83","first-page":"56274","article-title":"Rapid, Directed Differentiation of Retinal Pigment Epithelial Cells from Human Embryonic or Induced Pluripotent Stem Cells","volume":"128","author":"Foltz","year":"2017","journal-title":"J. Vis. Exp."},{"key":"10.1016\/j.omta.2026.201768_bib84","doi-asserted-by":"crossref","DOI":"10.1002\/0471140864.ps1403s62","article-title":"Analysis of protein prenylation in vitro and in vivo using functionalized phosphoisoprenoids","author":"Nguyen","year":"2010","journal-title":"Curr. Protoc. Protein Sci"},{"key":"10.1016\/j.omta.2026.201768_bib85","doi-asserted-by":"crossref","first-page":"ar116","DOI":"10.1091\/mbc.E24-11-0497","article-title":"Loss of REP-1 in retinal pigment epithelial cells leads to impaired phagosome processing and altered lysosomal pathway function","volume":"36","author":"Coelho","year":"2025","journal-title":"Mol. Biol. Cell"}],"container-title":["Molecular Therapy Advances"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S3117387X26001035?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S3117387X26001035?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T15:14:38Z","timestamp":1789053278000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S3117387X26001035"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,9]]},"references-count":85,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,9]]}},"alternative-id":["S3117387X26001035"],"URL":"https:\/\/doi.org\/10.1016\/j.omta.2026.201768","relation":{},"ISSN":["3117-387X"],"issn-type":[{"value":"3117-387X","type":"print"}],"subject":[],"published":{"date-parts":[[2026,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"mRNA delivery to the retina restores REP1 function in choroideremia","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Advances","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omta.2026.201768","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"201768"}}