{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,2]],"date-time":"2026-11-02T12:14:26Z","timestamp":1793621666912,"version":"4.5.0"},"publisher-location":"Cham","reference-count":24,"publisher":"Springer Nature Switzerland","isbn-type":[{"value":"9783031765490","type":"print"},{"value":"9783031765506","type":"electronic"}],"license":[{"start":{"date-parts":[[2025,1,1]],"date-time":"2025-01-01T00:00:00Z","timestamp":1735689600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2025,1,1]],"date-time":"2025-01-01T00:00:00Z","timestamp":1735689600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025]]},"DOI":"10.1007\/978-3-031-76550-6_20","type":"book-chapter","created":{"date-parts":[[2025,2,10]],"date-time":"2025-02-10T20:46:46Z","timestamp":1739220406000},"page":"119-123","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Precise Gene Editing Technologies in Retinal Applications"],"prefix":"10.1007","author":[{"given":"Mehri","family":"Ahmadian","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Iskalen Cansu Topcu","family":"Okan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gokce","family":"Uyanik","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Markus","family":"Tschopp","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cavit","family":"Agca","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,2,11]]},"reference":[{"key":"20_CR1","doi-asserted-by":"publisher","first-page":"aaf5573","DOI":"10.1126\/science.aaf5573","volume":"353","author":"OO Abudayyeh","year":"2016","unstructured":"Abudayyeh OO, Gootenberg JS, Konermann S, et al. C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector. Science. 2016;353:aaf5573.","journal-title":"Science"},{"key":"20_CR2","doi-asserted-by":"publisher","first-page":"924917","DOI":"10.3389\/fnins.2022.924917","volume":"16","author":"HY Altay","year":"2022","unstructured":"Altay HY, Ozdemir F, Afghah F, et al. Gene regulatory and gene editing tools and their applications for retinal diseases and neuroprotection: from proof-of-concept to clinical trial. Front Neurol. 2022;16:924917.","journal-title":"Front Neurol"},{"key":"20_CR3","doi-asserted-by":"publisher","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","volume":"576","author":"AV Anzalone","year":"2019","unstructured":"Anzalone AV, Randolph PB, Davis JR, et al. Search-and-replace genome editing without double-strand breaks or donor DNA. Nature. 2019;576:149\u201357.","journal-title":"Nature"},{"key":"20_CR4","doi-asserted-by":"publisher","first-page":"eabl9238","DOI":"10.1126\/scitranslmed.abl9238","volume":"14","author":"D Bock","year":"2022","unstructured":"Bock D, Rothgangl T, Villiger L, et al. In vivo prime editing of a metabolic liver disease in mice. Sci Transl Med. 2022;14:eabl9238.","journal-title":"Sci Transl Med"},{"key":"20_CR5","doi-asserted-by":"publisher","first-page":"1830","DOI":"10.1038\/s41467-022-29490-3","volume":"13","author":"EH Choi","year":"2022","unstructured":"Choi EH, Suh S, Foik AT, et al. In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration. Nat Commun. 2022;13:1830.","journal-title":"Nat Commun"},{"key":"20_CR6","doi-asserted-by":"publisher","first-page":"1019","DOI":"10.1126\/science.aaq0180","volume":"358","author":"DBT Cox","year":"2017","unstructured":"Cox DBT, Gootenberg JS, Abudayyeh OO, et al. RNA editing with CRISPR-Cas13. Science. 2017;358:1019\u201327.","journal-title":"Science"},{"key":"20_CR7","doi-asserted-by":"publisher","first-page":"58","DOI":"10.1016\/j.oret.2021.03.016","volume":"6","author":"WS Gange","year":"2022","unstructured":"Gange WS, Sisk RA, Besirli CG, et al. Perifoveal chorioretinal atrophy after subretinal voretigene neparvovec-rzyl for RPE65-mediated leber congenital amaurosis. Ophthalmol Retina. 2022;6:58\u201364.","journal-title":"Ophthalmol Retina"},{"key":"20_CR8","doi-asserted-by":"publisher","first-page":"464","DOI":"10.1038\/nature24644","volume":"551","author":"NM Gaudelli","year":"2017","unstructured":"Gaudelli NM, Komor AC, Rees HA, et al. Programmable base editing of T to G C in genomic DNA without DNA cleavage. Nature. 2017;551:464\u201371.","journal-title":"Nature"},{"key":"20_CR9","doi-asserted-by":"publisher","first-page":"9580","DOI":"10.1093\/nar\/gkac713","volume":"50","author":"W Han","year":"2022","unstructured":"Han W, Huang W, Wei T, et al. Programmable RNA base editing with a single gRNA-free enzyme. Nucleic Acids Res. 2022;50:9580\u201395.","journal-title":"Nucleic Acids Res"},{"key":"20_CR10","doi-asserted-by":"publisher","first-page":"181","DOI":"10.1038\/s41551-021-00788-9","volume":"6","author":"H Jang","year":"2022","unstructured":"Jang H, Jo DH, Cho CS, et al. Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases. Nat Biomed Eng. 2022;6:181\u201394.","journal-title":"Nat Biomed Eng"},{"key":"20_CR11","doi-asserted-by":"publisher","first-page":"816","DOI":"10.1126\/science.1225829","volume":"337","author":"M Jinek","year":"2012","unstructured":"Jinek M, Chylinski K, Fonfara I, et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012;337:816\u201321.","journal-title":"Science"},{"key":"20_CR12","doi-asserted-by":"publisher","first-page":"16","DOI":"10.1016\/j.omtn.2022.11.021","volume":"31","author":"DH Jo","year":"2023","unstructured":"Jo DH, Jang HK, Cho CS, et al. Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing. Mol Ther Nucleic Acids. 2023;31:16\u201327.","journal-title":"Mol Ther Nucleic Acids"},{"key":"20_CR13","doi-asserted-by":"publisher","first-page":"420","DOI":"10.1038\/nature17946","volume":"533","author":"AC Komor","year":"2016","unstructured":"Komor AC, Kim YB, Packer MS, et al. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature. 2016;533:420\u20134.","journal-title":"Nature"},{"key":"20_CR15","doi-asserted-by":"publisher","first-page":"144","DOI":"10.1038\/s41551-020-00656-y","volume":"5","author":"S Ling","year":"2021","unstructured":"Ling S, Yang S, Hu X, et al. Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice. Nat Biomed Eng. 2021;5:144\u201356.","journal-title":"Nat Biomed Eng"},{"key":"20_CR16","doi-asserted-by":"publisher","first-page":"161","DOI":"10.1016\/j.ophtha.2023.09.006","volume":"131","author":"B Lorenz","year":"2023","unstructured":"Lorenz B, K\u00fcnzel SH, Preising MN, et al. Real-world experience with Voretigene Neparvovec gene augmentation therapy in RPE65-mutation associated inherited retinal degeneration. Ophthalmology. 2023;131:161.","journal-title":"Ophthalmology"},{"key":"20_CR17","doi-asserted-by":"publisher","first-page":"229","DOI":"10.1038\/s41591-018-0327-9","volume":"25","author":"ML Maeder","year":"2019","unstructured":"Maeder ML, Stefanidakis M, Wilson CJ, et al. Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10. Nat Med. 2019;25:229\u201333.","journal-title":"Nat Med"},{"key":"20_CR18","first-page":"1460","volume-title":"Ophthalmology","author":"AM Maguire","year":"2021","unstructured":"Maguire AM, Russell S, Chung DC, et al. Durability of voretigene neparvovec for biallelic RPE65-mediated inherited retinal disease: phase 3 results at 3 and 4 years. In: Ophthalmology. 10th ed. Elsevier Inc; 2021. p. 1460\u20138.","edition":"10"},{"key":"20_CR19","doi-asserted-by":"publisher","unstructured":"Muller A, Sullivan J, Schwarzer W, et al. High-efficiency base editing for Stargardt disease in mice, non-human primates, and human retina tissue. bioRxiv. 2023; https:\/\/doi.org\/10.1101\/2023.04.17.535579.","DOI":"10.1101\/2023.04.17.535579"},{"key":"20_CR20","doi-asserted-by":"publisher","first-page":"759","DOI":"10.1038\/s41587-021-01105-0","volume":"40","author":"P Reautschnig","year":"2022","unstructured":"Reautschnig P, Wahn N, Wettengel J, et al. CLUSTER guide RNAs enable precise and efficient RNA editing with endogenous ADAR enzymes in vivo. Nat Biotechnol. 2022;40:759\u201368.","journal-title":"Nat Biotechnol"},{"key":"20_CR21","doi-asserted-by":"publisher","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","volume":"390","author":"S Russell","year":"2017","unstructured":"Russell S, Bennett J, Wellman JA, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017;390:849\u201360.","journal-title":"Lancet"},{"key":"20_CR22","doi-asserted-by":"publisher","first-page":"a017111","DOI":"10.1101\/cshperspect.a017111","volume":"5","author":"JA Sahel","year":"2015","unstructured":"Sahel JA, Marazova K, Audo I. Clinical characteristics and current therapies for inherited retinal degenerations. Cold Spring Harb Perspect Med. 2015;5:a017111.","journal-title":"Cold Spring Harb Perspect Med"},{"key":"20_CR23","doi-asserted-by":"publisher","first-page":"57","DOI":"10.1038\/s41392-022-01234-1","volume":"8","author":"K She","year":"2023","unstructured":"She K, Liu Y, Zhao Q, et al. Dual-AAV split prime editor corrects the mutation and phenotype in mice with inherited retinal degeneration. Signal Transduct Target Ther. 2023;8:57.","journal-title":"Signal Transduct Target Ther"},{"key":"20_CR24","doi-asserted-by":"publisher","first-page":"596","DOI":"10.1016\/j.omtn.2023.02.011","volume":"31","author":"J Su","year":"2023","unstructured":"Su J, She K, Song L, et al. In vivo base editing rescues photoreceptors in a mouse model of retinitis pigmentosa. Mol Ther Nucleic Acids. 2023;31:596\u2013609.","journal-title":"Mol Ther Nucleic Acids"},{"key":"20_CR25","doi-asserted-by":"publisher","first-page":"169","DOI":"10.1038\/s41551-020-00632-6","volume":"5","author":"S Suh","year":"2021","unstructured":"Suh S, Choi EH, Leinonen H, et al. Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing. Nat Biomed Eng. 2021;5:169\u201378.","journal-title":"Nat Biomed Eng"}],"container-title":["Advances in Experimental Medicine and Biology","Retinal Degenerative Diseases XX"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/978-3-031-76550-6_20","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,2,10]],"date-time":"2025-02-10T20:46:47Z","timestamp":1739220407000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/978-3-031-76550-6_20"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025]]},"ISBN":["9783031765490","9783031765506"],"references-count":24,"URL":"https:\/\/doi.org\/10.1007\/978-3-031-76550-6_20","relation":{},"ISSN":["0065-2598","2214-8019"],"issn-type":[{"value":"0065-2598","type":"print"},{"value":"2214-8019","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025]]},"assertion":[{"value":"11 February 2025","order":1,"name":"first_online","label":"First Online","group":{"name":"ChapterHistory","label":"Chapter History"}},{"value":"IWAAL","order":1,"name":"conference_acronym","label":"Conference Acronym","group":{"name":"ConferenceInfo","label":"Conference Information"}},{"value":"International Workshop on Ambient Assisted Living","order":2,"name":"conference_name","label":"Conference Name","group":{"name":"ConferenceInfo","label":"Conference Information"}},{"value":"Torremolinos","order":3,"name":"conference_city","label":"Conference City","group":{"name":"ConferenceInfo","label":"Conference Information"}},{"value":"Spain","order":4,"name":"conference_country","label":"Conference Country","group":{"name":"ConferenceInfo","label":"Conference Information"}},{"value":"2011","order":5,"name":"conference_year","label":"Conference Year","group":{"name":"ConferenceInfo","label":"Conference Information"}},{"value":"8 June 2011","order":7,"name":"conference_start_date","label":"Conference Start Date","group":{"name":"ConferenceInfo","label":"Conference Information"}},{"value":"10 June 2011","order":8,"name":"conference_end_date","label":"Conference End Date","group":{"name":"ConferenceInfo","label":"Conference Information"}},{"value":"3","order":9,"name":"conference_number","label":"Conference Number","group":{"name":"ConferenceInfo","label":"Conference Information"}},{"value":"7215","order":10,"name":"conference_id","label":"Conference ID","group":{"name":"ConferenceInfo","label":"Conference Information"}}]}}