{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,24]],"date-time":"2026-10-24T08:02:25Z","timestamp":1792828945365,"version":"4.5.0"},"reference-count":35,"publisher":"Elsevier BV","issue":"12","license":[{"start":{"date-parts":[[2023,12,1]],"date-time":"2023-12-01T00:00:00Z","timestamp":1701388800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,12,1]],"date-time":"2023-12-01T00:00:00Z","timestamp":1701388800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,12,6]],"date-time":"2024-12-06T00:00:00Z","timestamp":1733443200000},"content-version":"vor","delay-in-days":371,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100020737","name":"Shanghai Key Laboratory of Translational Medicine on Ear and Nose Diseases","doi-asserted-by":"publisher","award":["14DZ2260300"],"award-info":[{"award-number":["14DZ2260300"]}],"id":[{"id":"10.13039\/100020737","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81970872"],"award-info":[{"award-number":["81970872"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["82122019"],"award-info":[{"award-number":["82122019"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003399","name":"Science and Technology Commission of Shanghai Municipality","doi-asserted-by":"publisher","award":["21JC1404000"],"award-info":[{"award-number":["21JC1404000"]}],"id":[{"id":"10.13039\/501100003399","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2023,12]]},"DOI":"10.1016\/j.ymthe.2023.10.019","type":"journal-article","created":{"date-parts":[[2023,11,2]],"date-time":"2023-11-02T14:18:15Z","timestamp":1698934695000},"page":"3520-3530","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":56,"title":["RNA base editing therapy cures hearing loss induced by OTOF gene mutation"],"prefix":"10.1016","volume":"31","author":[{"given":"Yuanyuan","family":"Xue","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yong","family":"Tao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xing","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xueling","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yilai","family":"Shu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuanhua","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wen","family":"Kang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sifan","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhenzhe","family":"Cheng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Boou","family":"Yan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanwei","family":"Xie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lanting","family":"Bi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Haitao","family":"Jia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jinhui","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qingquan","family":"Xiao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liying","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xuan","family":"Yao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linyu","family":"Shi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8023-0230","authenticated-orcid":false,"given":"Hui","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hao","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2023.10.019_bib1","doi-asserted-by":"crossref","first-page":"2151","DOI":"10.1056\/NEJMra050700","article-title":"Newborn hearing screening--a silent revolution","volume":"354","author":"Morton","year":"2006","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2023.10.019_bib2","doi-asserted-by":"crossref","first-page":"996","DOI":"10.1016\/S0140-6736(21)00516-X","article-title":"Hearing loss prevalence and years lived with disability, 1990-2019: findings from the Global Burden of Disease Study 2019","volume":"397","year":"2021","journal-title":"Lancet"},{"key":"10.1016\/j.ymthe.2023.10.019_bib3","doi-asserted-by":"crossref","first-page":"R65","DOI":"10.1093\/hmg\/ddz129","article-title":"Gene therapy for hearing loss","volume":"28","author":"Omichi","year":"2019","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2023.10.019_bib4","doi-asserted-by":"crossref","first-page":"853","DOI":"10.1007\/s00439-021-02340-w","article-title":"The natural history of OTOF-related auditory neuropathy spectrum disorders: a multicenter study","volume":"141","author":"Thorpe","year":"2022","journal-title":"Hum. Genet."},{"key":"10.1016\/j.ymthe.2023.10.019_bib5","doi-asserted-by":"crossref","first-page":"363","DOI":"10.1038\/7693","article-title":"A mutation in OTOF, encoding otoferlin, a FER-1-like protein, causes DFNB9, a nonsyndromic form of deafness","volume":"21","author":"Yasunaga","year":"1999","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2023.10.019_bib6","doi-asserted-by":"crossref","first-page":"869","DOI":"10.1038\/nn.2578","article-title":"Hearing requires otoferlin-dependent efficient replenishment of synaptic vesicles in hair cells","volume":"13","author":"Pangrsic","year":"2010","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.ymthe.2023.10.019_bib7","doi-asserted-by":"crossref","first-page":"502","DOI":"10.1136\/jmg.39.7.502","article-title":"Q829X, a novel mutation in the gene encoding otoferlin (OTOF), is frequently found in Spanish patients with prelingual non-syndromic hearing loss","volume":"39","author":"Migliosi","year":"2002","journal-title":"J.\u00a0Med. Genet."},{"key":"10.1016\/j.ymthe.2023.10.019_bib8","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1682\/JRRD.2007.10.0173","article-title":"Cochlear implants: current designs and future possibilities","volume":"45","author":"Wilson","year":"2008","journal-title":"J.\u00a0Rehabil. Res. Dev."},{"key":"10.1016\/j.ymthe.2023.10.019_bib9","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1177\/108471380400800203","article-title":"Music perception with cochlear implants: a review","volume":"8","author":"McDermott","year":"2004","journal-title":"Trends Amplif."},{"key":"10.1016\/j.ymthe.2023.10.019_bib10","doi-asserted-by":"crossref","DOI":"10.1016\/j.heares.2022.108624","article-title":"Animal Models of Hearing Loss after Cochlear Implantation and Electrical Stimulation","volume":"426","author":"Reiss","year":"2022","journal-title":"Hear. Res."},{"key":"10.1016\/j.ymthe.2023.10.019_bib11","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1016\/j.ijporl.2017.01.020","article-title":"The temporoparietal fascia flap for coverage of cochlear reimplantation following extrusion","volume":"94","author":"Karimnejad","year":"2017","journal-title":"Int. J. Pediatr. Otorhinolaryngol."},{"key":"10.1016\/j.ymthe.2023.10.019_bib12","doi-asserted-by":"crossref","first-page":"4496","DOI":"10.1073\/pnas.1817537116","article-title":"Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model","volume":"116","author":"Akil","year":"2019","journal-title":"Proc. Natl. Acad. Sci. USA."},{"key":"10.1016\/j.ymthe.2023.10.019_bib13","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.201809396","article-title":"A dual-AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock-out mice","volume":"11","author":"Al-Moyed","year":"2019","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.ymthe.2023.10.019_bib14","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1007\/s00439-022-02504-2","article-title":"Hearing of Otof-deficient mice restored by trans-splicing of N- and C-terminal otoferlin","volume":"142","author":"Tang","year":"2023","journal-title":"Hum. Genet."},{"key":"10.1016\/j.ymthe.2023.10.019_bib15","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1038\/s41592-021-01124-4","article-title":"Programmable RNA editing with compact CRISPR-Cas13 systems from uncultivated microbes","volume":"18","author":"Xu","year":"2021","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2023.10.019_bib16","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.abn0449","article-title":"Rescue of autosomal dominant hearing loss by in\u00a0vivo delivery of mini dCas13X-derived RNA base editor","volume":"14","author":"Xiao","year":"2022","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2023.10.019_bib17","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1038\/s41592-019-0323-0","article-title":"In\u00a0vivo RNA editing of point mutations via RNA-guided adenosine deaminases","volume":"16","author":"Katrekar","year":"2019","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2023.10.019_bib18","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1126\/science.aax7063","article-title":"A cytosine deaminase for programmable single-base RNA editing","volume":"365","author":"Abudayyeh","year":"2019","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2023.10.019_bib19","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.ymthe.2023.10.019_bib20","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1016\/j.cell.2006.08.040","article-title":"Otoferlin, defective in a human deafness form, is essential for exocytosis at the auditory ribbon synapse","volume":"127","author":"Roux","year":"2006","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2023.10.019_bib21","first-page":"42","article-title":"Cochlea size variability and implications in clinical practice","volume":"34","author":"Pelliccia","year":"2014","journal-title":"Acta Otorhinolaryngol. Ital."},{"key":"10.1016\/j.ymthe.2023.10.019_bib22","doi-asserted-by":"crossref","first-page":"2242","DOI":"10.1121\/1.2180533","article-title":"Frequency- and level-dependent changes in auditory brainstem responses (ABRS) in developing mice","volume":"119","author":"Song","year":"2006","journal-title":"J.\u00a0Acoust. Soc. Am."},{"key":"10.1016\/j.ymthe.2023.10.019_bib23","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1016\/j.neuron.2012.05.019","article-title":"Restoration of hearing in the VGLUT3 knockout mouse using virally mediated gene therapy","volume":"75","author":"Akil","year":"2012","journal-title":"Neuron"},{"key":"10.1016\/j.ymthe.2023.10.019_bib24","doi-asserted-by":"crossref","first-page":"3382","DOI":"10.1172\/JCI94351","article-title":"Clarin-1 gene transfer rescues auditory synaptopathy in model of Usher syndrome","volume":"128","author":"Dulon","year":"2018","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2023.10.019_bib25","doi-asserted-by":"crossref","first-page":"780","DOI":"10.1016\/j.ymthe.2017.01.007","article-title":"Gene Therapy Restores Balance and Auditory Functions in a Mouse Model of Usher Syndrome","volume":"25","author":"Isgrig","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2023.10.019_bib26","doi-asserted-by":"crossref","first-page":"264","DOI":"10.1038\/nbt.3801","article-title":"Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c","volume":"35","author":"Pan","year":"2017","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2023.10.019_bib27","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.202013259","article-title":"Neonatal AAV gene therapy rescues hearing in a mouse model of SYNE4 deafness","volume":"13","author":"Taiber","year":"2021","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.ymthe.2023.10.019_bib28","doi-asserted-by":"crossref","first-page":"603","DOI":"10.1038\/gt.2015.27","article-title":"Potential treatments for genetic hearing loss in humans: current conundrums","volume":"22","author":"Minoda","year":"2015","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2023.10.019_bib29","doi-asserted-by":"crossref","first-page":"191","DOI":"10.1146\/annurev-biochem-013118-111730","article-title":"Evaluating and Enhancing Target Specificity of Gene-Editing Nucleases and Deaminases","volume":"88","author":"Kim","year":"2019","journal-title":"Annu. Rev. Biochem."},{"key":"10.1016\/j.ymthe.2023.10.019_bib30","doi-asserted-by":"crossref","first-page":"938","DOI":"10.1038\/s41587-021-01171-4","article-title":"Efficient in\u00a0vitro and in\u00a0vivo RNA editing via recruitment of endogenous ADARs using circular guide RNAs","volume":"40","author":"Katrekar","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2023.10.019_bib31","article-title":"Develop a Compact RNA Base Editor by Fusing ADAR with Engineered EcCas6e","volume":"10","author":"Wang","year":"2023","journal-title":"Adv. Sci."},{"key":"10.1016\/j.ymthe.2023.10.019_bib32","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1089\/crispr.2018.0014","article-title":"EditR: A Method to Quantify Base Editing from Sanger Sequencing","volume":"1","author":"Kluesner","year":"2018","journal-title":"CRISPR J."},{"key":"10.1016\/j.ymthe.2023.10.019_bib33","doi-asserted-by":"crossref","first-page":"224","DOI":"10.1038\/s41587-019-0032-3","article-title":"CRISPResso2 provides accurate and rapid genome editing sequence analysis","volume":"37","author":"Clement","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2023.10.019_bib34","doi-asserted-by":"crossref","first-page":"2286","DOI":"10.1016\/j.ymthe.2023.02.015","article-title":"A high-fidelity RNA-targeting Cas13 restores paternal Ube3a expression and improves motor functions in Angelman syndrome mice","volume":"31","author":"Li","year":"2023","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2023.10.019_bib35","doi-asserted-by":"crossref","DOI":"10.3390\/biom13010038","article-title":"Approaches and Vectors for Efficient Cochlear Gene Transfer in Adult Mouse Models","volume":"13","author":"Zhao","year":"2022","journal-title":"Biomolecules"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001623005968?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001623005968?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,26]],"date-time":"2025-10-26T18:11:17Z","timestamp":1761502277000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001623005968"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,12]]},"references-count":35,"journal-issue":{"issue":"12","published-print":{"date-parts":[[2023,12]]}},"alternative-id":["S1525001623005968"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2023.10.019","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2023,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"RNA base editing therapy cures hearing loss induced by OTOF gene mutation","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2023.10.019","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}