{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,24]],"date-time":"2026-10-24T08:02:29Z","timestamp":1792828949297,"version":"4.5.0"},"reference-count":143,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"funder":[{"DOI":"10.13039\/100019533","name":"Moderna Inc","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100019533","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Aspects of Medicine"],"published-print":{"date-parts":[[2026,2]]},"DOI":"10.1016\/j.mam.2025.101443","type":"journal-article","created":{"date-parts":[[2025,12,29]],"date-time":"2025-12-29T05:13:07Z","timestamp":1766985187000},"page":"101443","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":2,"special_numbering":"C","title":["The potential use of anti-codon engineered tRNAs (ACE-tRNAs) to treat nonsense variants causing inherited retinal diseases"],"prefix":"10.1016","volume":"107","author":[{"given":"Asodu Sandeep","family":"Sarma","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1789-5811","authenticated-orcid":false,"given":"Dror","family":"Sharon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.mam.2025.101443_bib1","article-title":"Preventing vision loss in a mouse model of Leber Congenital Amaurosis by engineered tRNA","author":"Akyuz","year":"2025","journal-title":"bioRxiv : The Preprint Server for Biology"},{"issue":"7966","key":"10.1016\/j.mam.2025.101443_bib2","doi-asserted-by":"crossref","first-page":"842","DOI":"10.1038\/s41586-023-06133-1","article-title":"Engineered tRNAs suppress nonsense mutations in cells and in vivo","volume":"618","author":"Albers","year":"2023","journal-title":"Nature"},{"issue":"4","key":"10.1016\/j.mam.2025.101443_bib3","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1208\/s12248-009-9143-y","article-title":"Nonviral gene delivery: principle, limitations, and recent progress","volume":"11","author":"Al-Dosari","year":"2009","journal-title":"AAPS J."},{"issue":"15","key":"10.1016\/j.mam.2025.101443_bib4","doi-asserted-by":"crossref","first-page":"2455","DOI":"10.1093\/hmg\/ddad072","article-title":"Inhibition of nonsense-mediated mRNA decay may improve stop codon read-through therapy for Duchenne muscular dystrophy","volume":"32","author":"Amar-Schwartz","year":"2023","journal-title":"Hum. Mol. Genet."},{"issue":"7785","key":"10.1016\/j.mam.2025.101443_bib5","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib6","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1353\/pbm.1970.0011","article-title":"The use of DNA for gene therapy--the need, experimental approach, and implications","volume":"14","author":"Aposhian","year":"1970","journal-title":"Perspect. Biol. Med."},{"issue":"6","key":"10.1016\/j.mam.2025.101443_bib7","doi-asserted-by":"crossref","DOI":"10.3390\/genes15060705","article-title":"Inherited retinal diseases and retinal organoids as preclinical cell models for inherited retinal disease research","volume":"15","author":"Ashworth","year":"2024","journal-title":"Genes"},{"issue":"5","key":"10.1016\/j.mam.2025.101443_bib8","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1136\/jmg.2006.045880","article-title":"Exon skipping through the creation of a putative exonic splicing silencer as a consequence of the cystic fibrosis mutation R553X","volume":"44","author":"Aznarez","year":"2007","journal-title":"J. Med. Genet."},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib9","doi-asserted-by":"crossref","first-page":"556","DOI":"10.1038\/mt.2015.220","article-title":"In vivo CRISPR\/Cas9 gene editing corrects retinal dystrophy in the S334ter-3 rat model of Autosomal dominant Retinitis pigmentosa","volume":"24","author":"Bakondi","year":"2016","journal-title":"Mol. Ther. : The Journal of the American Society of Gene Therapy"},{"issue":"23","key":"10.1016\/j.mam.2025.101443_bib10","doi-asserted-by":"crossref","first-page":"4767","DOI":"10.1093\/nar\/29.23.4767","article-title":"Misreading of termination codons in eukaryotes by natural nonsense suppressor tRNAs","volume":"29","author":"Beier","year":"2001","journal-title":"Nucleic Acids Res."},{"issue":"7","key":"10.1016\/j.mam.2025.101443_bib11","doi-asserted-by":"crossref","first-page":"1114","DOI":"10.1073\/pnas.48.7.1114","article-title":"A change from nonsense to sense in the genetic code","volume":"48","author":"Benzer","year":"1962","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib12","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-024-47258-9","article-title":"Translation velocity determines the efficacy of engineered suppressor tRNAs on pathogenic nonsense mutations","volume":"15","author":"Bharti","year":"2024","journal-title":"Nat. Commun."},{"issue":"15","key":"10.1016\/j.mam.2025.101443_bib13","doi-asserted-by":"crossref","first-page":"10061","DOI":"10.1093\/nar\/gku663","article-title":"New insights into the incorporation of natural suppressor tRNAs at stop codons in Saccharomyces cerevisiae","volume":"42","author":"Blanchet","year":"2014","journal-title":"Nucleic Acids Res."},{"issue":"12","key":"10.1016\/j.mam.2025.101443_bib14","doi-asserted-by":"crossref","first-page":"3018","DOI":"10.1073\/pnas.1715578115","article-title":"Deciphering the reading of the genetic code by near-cognate tRNA","volume":"115","author":"Blanchet","year":"2018","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"key":"10.1016\/j.mam.2025.101443_bib15","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtn.2023.102032","article-title":"Transfer RNA-mediated restoration of potassium current and electrical correction in premature termination long-QT syndrome hERG mutants","volume":"34","author":"Blomquist","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"issue":"11","key":"10.1016\/j.mam.2025.101443_bib16","article-title":"Antisense oligonucleotide mediated splice correction of a deep intronic mutation in OPA1","volume":"5","author":"Bonifert","year":"2016","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.mam.2025.101443_bib17","doi-asserted-by":"crossref","DOI":"10.1016\/j.preteyeres.2020.100915","article-title":"Immune responses to retinal gene therapy using adeno-associated viral vectors - implications for treatment success and safety","volume":"83","author":"Bucher","year":"2021","journal-title":"Prog. Retin. Eye Res."},{"issue":"6","key":"10.1016\/j.mam.2025.101443_bib18","doi-asserted-by":"crossref","first-page":"559","DOI":"10.1038\/sj.gt.3302653","article-title":"Potential applications for RNAi to probe pathogenesis and develop new treatments for ocular disorders","volume":"13","author":"Campochiaro","year":"2006","journal-title":"Gene Ther."},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib19","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1016\/0092-8674(75)90178-6","article-title":"Yeast super-suppressors are altered tRNAs capable of translating a nonsense codon in vitro","volume":"6","author":"Capecchi","year":"1975","journal-title":"Cell"},{"issue":"6","key":"10.1016\/j.mam.2025.101443_bib20","doi-asserted-by":"crossref","first-page":"1086","DOI":"10.1073\/pnas.48.6.1086","article-title":"On the role of soluble ribonucleic acid in coding for amino acids","volume":"48","author":"Chapeville","year":"1962","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"key":"10.1016\/j.mam.2025.101443_bib21","article-title":"Advances in the development of lipid nanoparticles for ophthalmic therapeutics","volume":"178","author":"Chen","year":"2024","journal-title":"Biomedicine & Pharmacotherapy = Biomedecine & Pharmacotherapie"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib22","doi-asserted-by":"crossref","DOI":"10.1038\/s41525-021-00180-1","article-title":"Genetic characteristics and epidemiology of inherited retinal degeneration in Taiwan","volume":"6","author":"Chen","year":"2021","journal-title":"NPJ Genomic Medicine"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib23","doi-asserted-by":"crossref","DOI":"10.1186\/s13287-023-03526-x","article-title":"Stem cell therapy for inherited retinal diseases: a systematic review and meta-analysis","volume":"14","author":"Chen","year":"2023","journal-title":"Stem Cell Res. Ther."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib24","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1023\/A:1005996125011","article-title":"Nonsense and missense translational suppression in plant cells mediated by tRNA(Lys)","volume":"36","author":"Chen","year":"1998","journal-title":"Plant Mol. Biol."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib25","doi-asserted-by":"crossref","DOI":"10.1186\/s12951-022-01717-x","article-title":"Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: applications, challenges, and emerging opportunities","volume":"20","author":"Chien","year":"2022","journal-title":"J. Nanobiotechnol."},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib26","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1016\/j.ajhg.2018.06.009","article-title":"Identifying Genes whose mutant transcripts cause dominant disease traits by potential gain-of-function alleles","volume":"103","author":"Coban-Akdemir","year":"2018","journal-title":"Am. J. Hum. Genet."},{"issue":"4","key":"10.1016\/j.mam.2025.101443_bib27","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1038\/gt.2014.8","article-title":"Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors","volume":"21","author":"Colella","year":"2014","journal-title":"Gene Ther."},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib28","doi-asserted-by":"crossref","first-page":"e14","DOI":"10.1038\/mtna.2012.3","article-title":"Antisense oligonucleotide (AON)-based therapy for Leber Congenital amaurosis caused by a frequent mutation in CEP290","volume":"1","author":"Collin","year":"2012","journal-title":"Mol. Ther. Nucleic Acids"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib29","doi-asserted-by":"crossref","DOI":"10.1038\/s42003-023-04555-1","article-title":"Lipid nanoparticles (LNP) induce activation and maturation of antigen presenting cells in young and aged individuals","volume":"6","author":"Connors","year":"2023","journal-title":"Commun. Biol."},{"key":"10.1016\/j.mam.2025.101443_bib30","article-title":"Identification of nonsense-mediated decay inhibitors that alter the tumor immune landscape","author":"Cook","year":"2024","journal-title":"bioRxiv : The Preprint Server for Biology"},{"key":"10.1016\/j.mam.2025.101443_bib31","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1016\/j.preteyeres.2017.10.004","article-title":"Molecular genetics and emerging therapies for retinitis pigmentosa: basic research and clinical perspectives","volume":"63","author":"Dias","year":"2018","journal-title":"Prog. Retin. Eye Res."},{"issue":"12","key":"10.1016\/j.mam.2025.101443_bib32","doi-asserted-by":"crossref","first-page":"2107","DOI":"10.1371\/journal.pgen.0020221","article-title":"Tissue-specific differences in human transfer RNA expression","volume":"2","author":"Dittmar","year":"2006","journal-title":"PLoS Genet."},{"issue":"7","key":"10.1016\/j.mam.2025.101443_bib33","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1167\/iovs.64.7.39","article-title":"Gene therapy for retinal degenerative diseases: progress, challenges, and future directions","volume":"64","author":"Drag","year":"2023","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib34","doi-asserted-by":"crossref","first-page":"166","DOI":"10.1089\/hgtb.2013.212","article-title":"Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A","volume":"25","author":"Dyka","year":"2014","journal-title":"Hum. Gene Ther. Methods"},{"key":"10.1016\/j.mam.2025.101443_bib35","doi-asserted-by":"crossref","DOI":"10.1016\/j.ebiom.2021.103515","article-title":"Translational readthrough of ciliopathy genes BBS2 and ALMS1 restores protein, ciliogenesis and function in patient fibroblasts","volume":"70","author":"Eintracht","year":"2021","journal-title":"EBioMedicine"},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib36","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1016\/j.molcel.2006.01.002","article-title":"Modified view of tRNA: stability amid sequence diversity","volume":"21","author":"Engelke","year":"2006","journal-title":"Mol. Cell"},{"issue":"6115","key":"10.1016\/j.mam.2025.101443_bib37","doi-asserted-by":"crossref","first-page":"808","DOI":"10.1038\/326808a0","article-title":"Role of arginine-tRNA in protein degradation by the ubiquitin pathway","volume":"326","author":"Ferber","year":"1987","journal-title":"Nature"},{"issue":"5563","key":"10.1016\/j.mam.2025.101443_bib38","doi-asserted-by":"crossref","first-page":"2258","DOI":"10.1126\/science.1067338","article-title":"An mRNA surveillance mechanism that eliminates transcripts lacking termination codons","volume":"295","author":"Frischmeyer","year":"2002","journal-title":"Science (New York, N.Y.)"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib39","doi-asserted-by":"crossref","DOI":"10.1038\/s42003-025-07557-3","article-title":"Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration","volume":"8","author":"Fry","year":"2025","journal-title":"Commun. Biol."},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib40","doi-asserted-by":"crossref","DOI":"10.3390\/ijms21030777","article-title":"RNA editing as a therapeutic approach for retinal gene therapy requiring long coding sequences","volume":"21","author":"Fry","year":"2020","journal-title":"Int. J. Mol. Sci."},{"issue":"7","key":"10.1016\/j.mam.2025.101443_bib41","doi-asserted-by":"crossref","first-page":"1121","DOI":"10.1073\/pnas.48.7.1121","article-title":"Suppression of mutations in the alkaline phosphatase structural cistron of E. coli","volume":"48","author":"Garen","year":"1962","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib42","doi-asserted-by":"crossref","first-page":"625","DOI":"10.1093\/genetics\/126.3.625","article-title":"Drosophila nonsense suppressors: functional analysis in Saccharomyces cerevisiae, Drosophila tissue culture cells and Drosophila melanogaster","volume":"126","author":"Garza","year":"1990","journal-title":"Genetics"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib43","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-023-42189-3","article-title":"Lipid nanoparticles with PEG-variant surface modifications mediate genome editing in the mouse retina","volume":"14","author":"Gautam","year":"2023","journal-title":"Nat. Commun."},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib44","doi-asserted-by":"crossref","first-page":"270","DOI":"10.1111\/ceo.13917","article-title":"Inherited retinal diseases: therapeutics, clinical trials and end points-A review","volume":"49","author":"Georgiou","year":"2021","journal-title":"Clin. Exp. Ophthalmol."},{"key":"10.1016\/j.mam.2025.101443_bib45","doi-asserted-by":"crossref","first-page":"1753","DOI":"10.2147\/OPTH.S364200","article-title":"Ocular gene therapy: a literature review with special focus on immune and inflammatory responses","volume":"16","author":"Ghoraba","year":"2022","journal-title":"Clin. Ophthalmol."},{"issue":"5","key":"10.1016\/j.mam.2025.101443_bib46","doi-asserted-by":"crossref","first-page":"761","DOI":"10.1093\/hmg\/ddx438","article-title":"Cas9\/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery","volume":"27","author":"Giannelli","year":"2018","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.mam.2025.101443_bib47","doi-asserted-by":"crossref","DOI":"10.1016\/j.ebiom.2025.105751","article-title":"Innovative therapies for inherited retinal dystrophies: navigating DNA, RNA, and protein approaches","volume":"116","author":"G\u00f3mez-Escribano","year":"2025","journal-title":"EBioMedicine"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib48","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1172\/JCI70462","article-title":"Postnatal manipulation of Pax 6 dosage reverses congenital tissue malformation defects","volume":"124","author":"Gregory-Evans","year":"2014","journal-title":"J. Clin. Investig."},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib49","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a041277","article-title":"Comparison of worldwide disease prevalence and genetic prevalence of inherited retinal diseases and variant interpretation considerations","volume":"14","author":"Hanany","year":"2024","journal-title":"Cold Spring Harb. Perspect. Med."},{"issue":"4","key":"10.1016\/j.mam.2025.101443_bib50","doi-asserted-by":"crossref","first-page":"521","DOI":"10.1038\/gt.2008.178","article-title":"Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injection","volume":"16","author":"Hellstr\u00f6m","year":"2009","journal-title":"Gene Ther."},{"issue":"16","key":"10.1016\/j.mam.2025.101443_bib51","doi-asserted-by":"crossref","first-page":"8421","DOI":"10.1073\/pnas.94.16.8421","article-title":"A Z-DNA binding domain present in the human editing enzyme, double-stranded RNA adenosine deaminase","volume":"94","author":"Herbert","year":"1997","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib52","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."},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib53","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1038\/nrm3270","article-title":"The unfolded protein response: controlling cell fate decisions under ER stress and beyond","volume":"13","author":"Hetz","year":"2012","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.mam.2025.101443_bib54","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1007\/978-1-4939-3271-9_2","article-title":"Delivering transgenic DNA exceeding the carrying capacity of AAV vectors","volume":"1382","author":"Hirsch","year":"2016","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.mam.2025.101443_bib55","doi-asserted-by":"crossref","DOI":"10.1016\/j.pharmthera.2025.108919","article-title":"Suppressor tRNAs as personalized therapy for nonsense mutation-associated pathologies","volume":"274","author":"Ignatova","year":"2025","journal-title":"Pharmacol. Ther."},{"issue":"5165","key":"10.1016\/j.mam.2025.101443_bib57","doi-asserted-by":"crossref","first-page":"1604","DOI":"10.1126\/science.8202715","article-title":"Digenic retinitis pigmentosa due to mutations at the unlinked peripherin\/RDS and ROM1 loci","volume":"264","author":"Kajiwara","year":"1994","journal-title":"Science"},{"key":"10.1016\/j.mam.2025.101443_bib58","doi-asserted-by":"crossref","first-page":"674","DOI":"10.1016\/j.omtn.2023.02.020","article-title":"Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C","volume":"31","author":"Kaltak","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.mam.2025.101443_bib59","doi-asserted-by":"crossref","DOI":"10.3389\/fgene.2022.858556","article-title":"Genetics of inherited retinal diseases in understudied populations","volume":"13","author":"Kannabiran","year":"2022","journal-title":"Front. Genet."},{"issue":"6","key":"10.1016\/j.mam.2025.101443_bib60","doi-asserted-by":"crossref","first-page":"938","DOI":"10.1038\/s41587-021-01171-4","article-title":"Efficient in vitro and in vivo RNA editing via recruitment of endogenous ADARs using circular guide RNAs","volume":"40","author":"Katrekar","year":"2022","journal-title":"Nat. Biotechnol."},{"issue":"5538","key":"10.1016\/j.mam.2025.101443_bib61","doi-asserted-by":"crossref","first-page":"2256","DOI":"10.1126\/science.1063525","article-title":"Triallelic inheritance in Bardet-Biedl syndrome, a Mendelian recessive disorder","volume":"293","author":"Katsanis","year":"2001","journal-title":"Science (New York, N.Y.)"},{"issue":"6","key":"10.1016\/j.mam.2025.101443_bib62","doi-asserted-by":"crossref","first-page":"2177","DOI":"10.1016\/S0002-9440(10)63267-1","article-title":"Inhibition of ocular angiogenesis by siRNA targeting vascular endothelial growth factor pathway genes: therapeutic strategy for herpetic stromal keratitis","volume":"165","author":"Kim","year":"2004","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.mam.2025.101443_bib63","doi-asserted-by":"crossref","first-page":"685","DOI":"10.1016\/j.omtn.2022.04.033","article-title":"Efficient suppression of endogenous CFTR nonsense mutations using anticodon-engineered transfer RNAs","volume":"28","author":"Ko","year":"2022","journal-title":"Mol. Ther. Nucleic Acids"},{"issue":"13","key":"10.1016\/j.mam.2025.101443_bib64","doi-asserted-by":"crossref","DOI":"10.1093\/nar\/gkaf675","article-title":"ACE-tRNAs are a platform technology for suppressing nonsense mutations that cause cystic fibrosis","volume":"53","author":"Ko","year":"2025","journal-title":"Nucleic Acids Res."},{"issue":"7603","key":"10.1016\/j.mam.2025.101443_bib65","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":"9","key":"10.1016\/j.mam.2025.101443_bib66","doi-asserted-by":"crossref","first-page":"2668","DOI":"10.1073\/pnas.84.9.2668","article-title":"Natural UAG suppressor glutamine tRNA is elevated in mouse cells infected with Moloney murine leukemia virus","volume":"84","author":"Kuchino","year":"1987","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"key":"10.1016\/j.mam.2025.101443_bib67","first-page":"245","article-title":"Methods, potentials, and limitations of gene delivery to regenerate central nervous system cells","volume":"3","author":"Kumar","year":"2009","journal-title":"Biol. Targets & Ther."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib68","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/s13023-021-02145-0","article-title":"Genetic testing and diagnosis of inherited retinal diseases","volume":"16","author":"Lam","year":"2021","journal-title":"Orphanet J. Rare Dis."},{"issue":"11","key":"10.1016\/j.mam.2025.101443_bib69","article-title":"In vivo editing of the human mutant rhodopsin gene by electroporation of plasmid-based CRISPR\/Cas9 in the mouse retina","volume":"5","author":"Latella","year":"2016","journal-title":"Mol. Ther. Nucleic Acids"},{"issue":"4","key":"10.1016\/j.mam.2025.101443_bib70","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1002\/jgm.1126","article-title":"Novel AAV serotypes for improved ocular gene transfer","volume":"10","author":"Lebherz","year":"2008","journal-title":"J. Gene Med."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib71","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1159\/000526317","article-title":"Gene therapy for inherited retinal disease: Long-Term durability of effect","volume":"66","author":"Leroy","year":"2023","journal-title":"Ophthalmic Res."},{"issue":"10","key":"10.1016\/j.mam.2025.101443_bib72","doi-asserted-by":"crossref","first-page":"2187","DOI":"10.1016\/j.stemcr.2022.08.005","article-title":"Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis","volume":"17","author":"Leung","year":"2022","journal-title":"Stem Cell Rep."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib73","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1089\/crispr.2017.0009","article-title":"Allele-Specific CRISPR-Cas9 genome editing of the single-base P23H mutation for rhodopsin-associated Dominant Retinitis pigmentosa","volume":"1","author":"Li","year":"2018","journal-title":"CRISPR J."},{"issue":"4","key":"10.1016\/j.mam.2025.101443_bib74","doi-asserted-by":"crossref","first-page":"324","DOI":"10.1038\/nbt.4102","article-title":"Base editing with a Cpf1-cytidine deaminase fusion","volume":"36","author":"Li","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.mam.2025.101443_bib75","doi-asserted-by":"crossref","first-page":"240","DOI":"10.1016\/j.omtn.2023.06.016","article-title":"Restoration of functional PAX6 in aniridia patient iPSC-derived ocular tissue models using repurposed nonsense suppression drugs","volume":"33","author":"Lima Cunha","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.mam.2025.101443_bib76","first-page":"1","article-title":"Translation termination codons in protein synthesis and disease","volume":"132","author":"Lombardi","year":"2022","journal-title":"Adv. Protein Chem. Struct. Biol."},{"issue":"8","key":"10.1016\/j.mam.2025.101443_bib77","doi-asserted-by":"crossref","first-page":"824","DOI":"10.1038\/gt.2013.3","article-title":"Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus","volume":"20","author":"Lopes","year":"2013","journal-title":"Gene Ther."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib78","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-019-08329-4","article-title":"Engineered transfer RNAs for suppression of premature termination codons","volume":"10","author":"Lueck","year":"2019","journal-title":"Nat. Commun."},{"issue":"11","key":"10.1016\/j.mam.2025.101443_bib79","doi-asserted-by":"crossref","first-page":"665","DOI":"10.1038\/nrm4063","article-title":"Nonsense-mediated mRNA decay: an intricate machinery that shapes transcriptomes","volume":"16","author":"Lykke-Andersen","year":"2015","journal-title":"Nat. Rev. Mol. Cell Biol."},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib80","doi-asserted-by":"crossref","first-page":"524","DOI":"10.1016\/j.ymthe.2017.11.019","article-title":"Triple vectors expand AAV transfer capacity in the retina","volume":"26","author":"Maddalena","year":"2018","journal-title":"Mol. Ther. : The Journal of the American Society of Gene Therapy"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib81","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-024-46703-z","article-title":"Extended stop codon context predicts nonsense codon readthrough efficiency in human cells","volume":"15","author":"Mangkalaphiban","year":"2024","journal-title":"Nat. Commun."},{"issue":"7","key":"10.1016\/j.mam.2025.101443_bib82","doi-asserted-by":"crossref","first-page":"1044","DOI":"10.1017\/S1355838200000716","article-title":"Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system","volume":"6","author":"Manuvakhova","year":"2000","journal-title":"RNA"},{"issue":"6","key":"10.1016\/j.mam.2025.101443_bib83","doi-asserted-by":"crossref","DOI":"10.1016\/S0960-9822(02)00747-9","article-title":"Nonsense-mediated mRNA decay","volume":"12","author":"Maquat","year":"2002","journal-title":"Curr. Biol. : Cailiao Baohu"},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib84","doi-asserted-by":"crossref","first-page":"368","DOI":"10.1016\/j.cell.2010.07.022","article-title":"The pioneer round of translation: features and functions","volume":"142","author":"Maquat","year":"2010","journal-title":"Cell"},{"issue":"11","key":"10.1016\/j.mam.2025.101443_bib85","doi-asserted-by":"crossref","first-page":"3104","DOI":"10.1158\/0008-5472.CAN-13-2235","article-title":"Identification and characterization of small molecules that inhibit nonsense-mediated RNA decay and suppress nonsense p53 mutations","volume":"74","author":"Martin","year":"2014","journal-title":"Cancer Res."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib86","doi-asserted-by":"crossref","first-page":"1","DOI":"10.3390\/ijms22010344","article-title":"Synergy between readthrough and nonsense mediated decay inhibition in a murine model of cystic fibrosis nonsense mutations","volume":"22","author":"McHugh","year":"2020","journal-title":"Int. J. Mol. Sci."},{"issue":"5","key":"10.1016\/j.mam.2025.101443_bib87","doi-asserted-by":"crossref","first-page":"668","DOI":"10.1016\/j.molcel.2010.01.023","article-title":"tRNA binds to cytochrome c and inhibits caspase activation","volume":"37","author":"Mei","year":"2010","journal-title":"Mol. Cell"},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib88","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1038\/s41587-019-0013-6","article-title":"Precise RNA editing by recruiting endogenous ADARs with antisense oligonucleotides","volume":"37","author":"Merkle","year":"2019","journal-title":"Nat. Biotechnol."},{"issue":"6","key":"10.1016\/j.mam.2025.101443_bib89","doi-asserted-by":"crossref","first-page":"633","DOI":"10.1177\/089686080602600603","article-title":"Gene transfer using nonviral delivery systems","volume":"26","author":"Miyazaki","year":"2006","journal-title":"Perit. Dial. Int. : Journal of the International Society for Peritoneal Dialysis"},{"issue":"7","key":"10.1016\/j.mam.2025.101443_bib90","doi-asserted-by":"crossref","first-page":"1093","DOI":"10.1038\/s41587-022-01225-1","article-title":"Endogenous ADAR-mediated RNA editing in non-human primates using stereopure chemically modified oligonucleotides","volume":"40","author":"Monian","year":"2022","journal-title":"Nat. Biotechnol."},{"issue":"8","key":"10.1016\/j.mam.2025.101443_bib91","doi-asserted-by":"crossref","first-page":"1037","DOI":"10.1002\/humu.20763","article-title":"A meta-analysis of nonsense mutations causing human genetic disease","volume":"29","author":"Mort","year":"2008","journal-title":"Hum. Mutat."},{"issue":"11","key":"10.1016\/j.mam.2025.101443_bib92","doi-asserted-by":"crossref","first-page":"6362","DOI":"10.1167\/iovs.15-16400","article-title":"Allele-Specific inhibition of rhodopsin with an antisense oligonucleotide slows photoreceptor cell degeneration","volume":"56","author":"Murray","year":"2015","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"issue":"6305","key":"10.1016\/j.mam.2025.101443_bib93","doi-asserted-by":"crossref","DOI":"10.1126\/science.aaf8729","article-title":"Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems","volume":"353","author":"Nishida","year":"2016","journal-title":"Science (New York, N.Y.)"},{"key":"10.1016\/j.mam.2025.101443_bib94","doi-asserted-by":"crossref","first-page":"1275","DOI":"10.1016\/j.msec.2017.04.068","article-title":"Non-viral strategies for ocular gene delivery","volume":"77","author":"Oliveira","year":"2017","journal-title":"Mater. Sci. Eng., C"},{"issue":"4","key":"10.1016\/j.mam.2025.101443_bib95","doi-asserted-by":"crossref","first-page":"669","DOI":"10.1016\/j.molcel.2025.01.026","article-title":"tRNA intron-derived small regulatory RNAs fine-tune gene expression under oxidative stress","volume":"85","author":"Pan","year":"2025","journal-title":"Mol. Cell"},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib96","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1016\/j.visres.2007.08.009","article-title":"Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: effects of serotype and site of administration","volume":"48","author":"Pang","year":"2008","journal-title":"Vis. Res."},{"key":"10.1016\/j.mam.2025.101443_bib97","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 : Official Journal of the Controlled Release Society"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib98","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-024-82766-0","article-title":"Engineered tRNAs efficiently suppress CDKL5 premature termination codons","volume":"14","author":"Pezzini","year":"2024","journal-title":"Sci. Rep."},{"issue":"9","key":"10.1016\/j.mam.2025.101443_bib99","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a017285","article-title":"The status of RPE65 gene therapy trials: safety and efficacy","volume":"5","author":"Pierce","year":"2015","journal-title":"Cold Spring Harb. Perspect. Med."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib100","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-020-17879-x","article-title":"Quantitative tRNA-sequencing uncovers metazoan tissue-specific tRNA regulation","volume":"11","author":"Pinkard","year":"2020","journal-title":"Nat. Commun."},{"issue":"4","key":"10.1016\/j.mam.2025.101443_bib101","doi-asserted-by":"crossref","DOI":"10.1002\/wrna.1641","article-title":"Therapeutic promise of engineered nonsense suppressor tRNAs","volume":"12","author":"Porter","year":"2021","journal-title":"Wiley Interdiscip. Rev. RNA"},{"issue":"22","key":"10.1016\/j.mam.2025.101443_bib102","doi-asserted-by":"crossref","first-page":"14112","DOI":"10.1093\/nar\/gkae1112","article-title":"Optimization of Anticodon edited transfer RNAs (ACE-tRNAs) function in translation for suppression of nonsense mutations","volume":"52","year":"2024","journal-title":"Nucleic Acids Res."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib103","doi-asserted-by":"crossref","DOI":"10.1183\/23120541.00080-2017","article-title":"Factors influencing readthrough therapy for frequent cystic fibrosis premature termination codons","volume":"4","author":"Pranke","year":"2018","journal-title":"ERJ Open Research"},{"issue":"9","key":"10.1016\/j.mam.2025.101443_bib104","doi-asserted-by":"crossref","first-page":"1059","DOI":"10.1038\/s41587-019-0178-z","article-title":"Programmable RNA editing by recruiting endogenous ADAR using engineered RNAs","volume":"37","author":"Qu","year":"2019","journal-title":"Nat. Biotechnol."},{"issue":"10","key":"10.1016\/j.mam.2025.101443_bib105","first-page":"E1941","article-title":"Systemic delivery of factor IX messenger RNA for protein replacement therapy","volume":"114","author":"Ramaswamy","year":"2017","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"issue":"5","key":"10.1016\/j.mam.2025.101443_bib106","doi-asserted-by":"crossref","first-page":"319","DOI":"10.4155\/tde-2016-0006","article-title":"mRNA vaccine delivery using lipid nanoparticles","volume":"7","author":"Reichmuth","year":"2016","journal-title":"Ther. Deliv."},{"key":"10.1016\/j.mam.2025.101443_bib107","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.ajhg.2025.08.017","article-title":"RetiGene, a comprehensive gene atlas for inherited retinal diseases","volume":"112","author":"Rivolta","year":"2025","journal-title":"AJHG"},{"issue":"44","key":"10.1016\/j.mam.2025.101443_bib108","doi-asserted-by":"crossref","first-page":"12508","DOI":"10.1073\/pnas.1605336113","article-title":"Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression","volume":"113","author":"Roy","year":"2016","journal-title":"Proc. Natl. Acad. Sci."},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib109","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1016\/j.ymthe.2016.12.006","article-title":"CRISPR\/Cas9-Mediated genome editing as a therapeutic approach for Leber Congenital amaurosis 10","volume":"25","author":"Ruan","year":"2017","journal-title":"Mol. Ther."},{"issue":"5","key":"10.1016\/j.mam.2025.101443_bib110","doi-asserted-by":"crossref","first-page":"1014","DOI":"10.1038\/s41591-022-01755-w","article-title":"Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b\/2 trial","volume":"28","author":"Russell","year":"2022","journal-title":"Nat. Med."},{"issue":"8","key":"10.1016\/j.mam.2025.101443_bib111","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pgen.1006264","article-title":"Tissue- and time-specific expression of otherwise identical tRNA genes","volume":"12","author":"Sagi","year":"2016","journal-title":"PLoS Genet."},{"issue":"12","key":"10.1016\/j.mam.2025.101443_bib112","doi-asserted-by":"crossref","first-page":"6104","DOI":"10.1128\/AAC.01326-12","article-title":"Structure-activity relationships among the kanamycin aminoglycosides: role of ring I hydroxyl and amino groups","volume":"56","author":"Salian","year":"2012","journal-title":"Antimicrob. Agents Chemother."},{"key":"10.1016\/j.mam.2025.101443_bib113","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."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib114","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtn.2024.102130","article-title":"A pipeline for identifying guide RNA sequences that promote RNA editing of nonsense mutations that cause inherited retinal diseases","volume":"35","author":"Schneider","year":"2024","journal-title":"Mol. Ther. Nucleic Acids"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib115","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-021-24575-x","article-title":"A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion","volume":"12","author":"Sharma","year":"2021","journal-title":"Nat. Commun."},{"issue":"44","key":"10.1016\/j.mam.2025.101443_bib116","doi-asserted-by":"crossref","first-page":"12353","DOI":"10.1073\/pnas.1615548113","article-title":"Proposing a mechanism of action for ataluren","volume":"113","author":"Siddiqui","year":"2016","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"issue":"10","key":"10.1016\/j.mam.2025.101443_bib117","article-title":"Antisense oligonucleotide-based splice correction for USH2A-associated retinal degeneration caused by a frequent deep-intronic mutation","volume":"5","author":"Slijkerman","year":"2016","journal-title":"Mol. Ther. Nucleic Acids"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib118","doi-asserted-by":"crossref","DOI":"10.1038\/s41439-022-00203-y","article-title":"A novel nonsense variant in ARID1B causing simultaneous RNA decay and exon skipping is associated with Coffin-Siris syndrome","volume":"9","author":"Sofronova","year":"2022","journal-title":"Human Genome Variation"},{"issue":"12","key":"10.1016\/j.mam.2025.101443_bib119","doi-asserted-by":"crossref","DOI":"10.1093\/nar\/gkaf532","article-title":"An engineered glutamic acid tRNA for efficient suppression of pathogenic nonsense mutations","volume":"53","author":"Specht","year":"2025","journal-title":"Nucleic Acids Res."},{"issue":"9","key":"10.1016\/j.mam.2025.101443_bib120","first-page":"1975","article-title":"Rapid ocular angiogenic control via naked DNA delivery to cornea","volume":"42","author":"Stechschulte","year":"2001","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"issue":"6","key":"10.1016\/j.mam.2025.101443_bib121","doi-asserted-by":"crossref","DOI":"10.3390\/genes15060804","article-title":"Genetic and clinical analyses of the KIZ-c.226C>T variant resulting in a dual mutational mechanism","volume":"15","author":"Sundaresan","year":"2024","journal-title":"Genes"},{"issue":"7","key":"10.1016\/j.mam.2025.101443_bib122","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1016\/j.tig.2020.11.002","article-title":"To NMD or not to NMD: Nonsense-Mediated mRNA decay in cancer and other genetic diseases","volume":"37","author":"Supek","year":"2021","journal-title":"Trends Genet. : TIG (Trends Genet.)"},{"key":"10.1016\/j.mam.2025.101443_bib123","doi-asserted-by":"crossref","DOI":"10.1093\/nar\/gkae624","article-title":"mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs)","author":"Susorov","year":"2024","journal-title":"Nucleic Acids Res."},{"issue":"10","key":"10.1016\/j.mam.2025.101443_bib124","doi-asserted-by":"crossref","DOI":"10.3390\/diagnostics10100779","article-title":"Syndromic inherited retinal diseases: genetic, clinical and diagnostic aspects","volume":"10","author":"Tatour","year":"2020","journal-title":"Diagnostics"},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib125","doi-asserted-by":"crossref","DOI":"10.3390\/biomedicines11030659","article-title":"Recoding of nonsense mutation as a pharmacological strategy","volume":"11","author":"Temaj","year":"2023","journal-title":"Biomedicines"},{"issue":"5857","key":"10.1016\/j.mam.2025.101443_bib126","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1038\/296537a0","article-title":"Construction of a functional human suppressor tRNA gene: an approach to gene therapy for beta-thalassaemia","volume":"296","author":"Temple","year":"1982","journal-title":"Nature"},{"issue":"13","key":"10.1016\/j.mam.2025.101443_bib127","doi-asserted-by":"crossref","first-page":"2504","DOI":"10.1038\/s41433-024-03065-6","article-title":"Voretigene neparvovec for inherited retinal dystrophy due to RPE65 mutations: a scoping review of eligibility and treatment challenges from clinical trials to real practice","volume":"38","author":"Testa","year":"2024","journal-title":"Eye (London, England)"},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib128","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1101\/gr.256164.119","article-title":"Predicting transfer RNA gene activity from sequence and genome context","volume":"30","author":"Thornlow","year":"2020","journal-title":"Genome Res."},{"issue":"9","key":"10.1016\/j.mam.2025.101443_bib129","doi-asserted-by":"crossref","first-page":"2755","DOI":"10.1016\/j.ymthe.2023.06.012","article-title":"Successful large gene augmentation of USH2A with non-viral episomal vectors","volume":"31","author":"Toms","year":"2023","journal-title":"Mol. Ther. : The Journal of the American Society of Gene Therapy"},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib130","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1002\/emmm.201302948","article-title":"Effective delivery of large genes to the retina by dual AAV vectors","volume":"6","author":"Trapani","year":"2014","journal-title":"EMBO Mol. Med."},{"issue":"9","key":"10.1016\/j.mam.2025.101443_bib131","doi-asserted-by":"crossref","first-page":"1421","DOI":"10.1016\/j.ophtha.2018.04.001","article-title":"Clustered regularly interspaced short palindromic repeats-based genome surgery for the treatment of Autosomal dominant Retinitis pigmentosa","volume":"125","author":"Tsai","year":"2018","journal-title":"Ophthalmology"},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib132","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1016\/j.ymthe.2006.04.011","article-title":"Specific inhibition of nonsense-mediated mRNA decay components, SMG-1 or Upf1, rescues the phenotype of Ullrich disease fibroblasts","volume":"14","author":"Usuki","year":"2006","journal-title":"Mol. Ther. : The Journal of the American Society of Gene Therapy"},{"issue":"7905","key":"10.1016\/j.mam.2025.101443_bib133","doi-asserted-by":"crossref","first-page":"343","DOI":"10.1038\/s41586-022-04533-3","article-title":"AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice","volume":"604","author":"Wang","year":"2022","journal-title":"Nature"},{"issue":"4","key":"10.1016\/j.mam.2025.101443_bib134","doi-asserted-by":"crossref","DOI":"10.1016\/j.neurot.2024.e00391","article-title":"Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders","volume":"21","author":"Wang","year":"2024","journal-title":"Neurotherapeutics : The Journal of the American Society for Experimental NeuroTherapeutics"},{"issue":"3","key":"10.1016\/j.mam.2025.101443_bib135","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtn.2024.102236","article-title":"Lipid nanoparticle technology-mediated therapeutic gene manipulation in the eyes","volume":"35","author":"Wang","year":"2024","journal-title":"Mol. Ther. Nucleic Acids"},{"issue":"4","key":"10.1016\/j.mam.2025.101443_bib136","doi-asserted-by":"crossref","first-page":"880","DOI":"10.1093\/emboj\/20.4.880","article-title":"The role of Upf proteins in modulating the translation read-through of nonsense-containing transcripts","volume":"20","author":"Wang","year":"2001","journal-title":"EMBO J."},{"key":"10.1016\/j.mam.2025.101443_bib137","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.52611","article-title":"Stop codon context influences genome-wide stimulation of termination codon readthrough by aminoglycosides","volume":"9","author":"Wangen","year":"2020","journal-title":"eLife"},{"issue":"7140","key":"10.1016\/j.mam.2025.101443_bib138","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1038\/nature05756","article-title":"PTC124 targets genetic disorders caused by nonsense mutations","volume":"447","author":"Welch","year":"2007","journal-title":"Nature"},{"issue":"2","key":"10.1016\/j.mam.2025.101443_bib139","doi-asserted-by":"crossref","first-page":"575","DOI":"10.1016\/0092-8674(83)90438-5","article-title":"The genes sup-7 X and sup-5 III of C. elegans suppress amber nonsense mutations via altered transfer RNA","volume":"33","author":"Wills","year":"1983","journal-title":"Cell"},{"key":"10.1016\/j.mam.2025.101443_bib140","doi-asserted-by":"crossref","first-page":"750","DOI":"10.1016\/j.omtn.2023.08.002","article-title":"Treatment of autosomal dominant retinitis pigmentosa caused by RHO-P23H mutation with high-fidelity Cas13X in mice","volume":"33","author":"Yan","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"issue":"R1","key":"10.1016\/j.mam.2025.101443_bib141","doi-asserted-by":"crossref","first-page":"R12","DOI":"10.1093\/hmg\/ddx182","article-title":"Inherited eye-related disorders due to mitochondrial dysfunction","volume":"26","author":"Yu-Wai-Man","year":"2017","journal-title":"Hum. Mol. Genet."},{"issue":"10","key":"10.1016\/j.mam.2025.101443_bib142","doi-asserted-by":"crossref","DOI":"10.3390\/vaccines11101580","article-title":"The expression kinetics and immunogenicity of lipid nanoparticles delivering plasmid DNA and mRNA in mice","volume":"11","author":"Zhang","year":"2023","journal-title":"Vaccines"},{"issue":"8","key":"10.1016\/j.mam.2025.101443_bib143","doi-asserted-by":"crossref","first-page":"1000","DOI":"10.1016\/j.tibtech.2023.03.004","article-title":"Prime editing: advances and therapeutic applications","volume":"41","author":"Zhao","year":"2023","journal-title":"Trends Biotechnol."},{"issue":"1","key":"10.1016\/j.mam.2025.101443_bib144","article-title":"Ocular immune privilege","volume":"2","author":"Zhou","year":"2010","journal-title":"F1000 Biology Reports"}],"container-title":["Molecular Aspects of Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0098299725001074?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0098299725001074?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,1,21]],"date-time":"2026-01-21T22:07:57Z","timestamp":1769033277000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0098299725001074"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,2]]},"references-count":143,"alternative-id":["S0098299725001074"],"URL":"https:\/\/doi.org\/10.1016\/j.mam.2025.101443","relation":{},"ISSN":["0098-2997"],"issn-type":[{"value":"0098-2997","type":"print"}],"subject":[],"published":{"date-parts":[[2026,2]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"The potential use of anti-codon engineered tRNAs (ACE-tRNAs) to treat nonsense variants causing inherited retinal diseases","name":"articletitle","label":"Article Title"},{"value":"Molecular Aspects of Medicine","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.mam.2025.101443","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 Elsevier Ltd. All rights are reserved, including those for text and data mining, AI training, and similar technologies.","name":"copyright","label":"Copyright"}],"article-number":"101443"}}