{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,5]],"date-time":"2026-11-05T04:11:18Z","timestamp":1793851878649,"version":"4.6.1"},"reference-count":61,"publisher":"Springer Science and Business Media LLC","issue":"7905","license":[{"start":{"date-parts":[[2022,3,23]],"date-time":"2022-03-23T00:00:00Z","timestamp":1647993600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2022,3,23]],"date-time":"2022-03-23T00:00:00Z","timestamp":1647993600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Nature"],"published-print":{"date-parts":[[2022,4,14]]},"DOI":"10.1038\/s41586-022-04533-3","type":"journal-article","created":{"date-parts":[[2022,3,23]],"date-time":"2022-03-23T17:03:37Z","timestamp":1648055017000},"page":"343-348","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":147,"title":["AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice"],"prefix":"10.1038","volume":"604","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4834-967X","authenticated-orcid":false,"given":"Jiaming","family":"Wang","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1144-1445","authenticated-orcid":false,"given":"Yue","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9938-6481","authenticated-orcid":false,"given":"Craig A.","family":"Mendonca","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Onur","family":"Yukselen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Khaja","family":"Muneeruddin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lingzhi","family":"Ren","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jialing","family":"Liang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chen","family":"Zhou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9565-1567","authenticated-orcid":false,"given":"Jun","family":"Xie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jia","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhong","family":"Jiang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9983-394X","authenticated-orcid":false,"given":"Alper","family":"Kucukural","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Scott A.","family":"Shaffer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0097-9012","authenticated-orcid":false,"given":"Guangping","family":"Gao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9079-2360","authenticated-orcid":false,"given":"Dan","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2022,3,23]]},"reference":[{"key":"4533_CR1","doi-asserted-by":"publisher","first-page":"358","DOI":"10.1038\/s41573-019-0012-9","volume":"18","author":"D Wang","year":"2019","unstructured":"Wang, D., Tai, P. W. L. & Gao, G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat. Rev. Drug Discov. 18, 358\u2013378 (2019).","journal-title":"Nat. Rev. Drug Discov."},{"key":"4533_CR2","doi-asserted-by":"publisher","first-page":"255","DOI":"10.1038\/s41576-019-0205-4","volume":"21","author":"C Li","year":"2020","unstructured":"Li, C. & Samulski, R. J. Engineering adeno-associated virus vectors for gene therapy. Nat. Rev. Genet. 21, 255\u2013272 (2020).","journal-title":"Nat. Rev. Genet."},{"key":"4533_CR3","doi-asserted-by":"publisher","first-page":"464","DOI":"10.1016\/j.ymthe.2020.12.007","volume":"29","author":"JR Mendell","year":"2020","unstructured":"Mendell, J. R. et al. Current clinical applications of in vivo gene therapy with AAVs. Mol. Ther. 29, 464\u2013488 (2020).","journal-title":"Mol. Ther."},{"key":"4533_CR4","doi-asserted-by":"publisher","first-page":"808","DOI":"10.1089\/hum.2020.167","volume":"31","author":"J Hordeaux","year":"2020","unstructured":"Hordeaux, J. et al. Adeno-associated virus-induced dorsal root ganglion pathology. Hum. Gene Ther. 31, 808\u2013818 (2020).","journal-title":"Hum. Gene Ther."},{"key":"4533_CR5","doi-asserted-by":"publisher","first-page":"930","DOI":"10.1038\/s41593-021-00827-3","volume":"24","author":"M Van Alstyne","year":"2021","unstructured":"Van Alstyne, M. et al. Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit. Nat. Neurosci. 24, 930\u2013940 (2021).","journal-title":"Nat. Neurosci."},{"key":"4533_CR6","doi-asserted-by":"publisher","first-page":"510","DOI":"10.1089\/hum.2016.109","volume":"28","author":"D Golebiowski","year":"2017","unstructured":"Golebiowski, D. et al. Direct intracranial injection of AAVrh8 encoding monkey \u03b2-N-acetylhexosaminidase causes neurotoxicity in the primate brain. Hum. Gene Ther. 28, 510\u2013522 (2017).","journal-title":"Hum. Gene Ther."},{"key":"4533_CR7","doi-asserted-by":"publisher","first-page":"136","DOI":"10.1016\/j.cell.2020.03.023","volume":"181","author":"D Wang","year":"2020","unstructured":"Wang, D., Zhang, F. & Gao, G. CRISPR-based therapeutic genome editing: strategies and in vivo delivery by AAV vectors. Cell\u00a0181, 136\u2013150 (2020).","journal-title":"Cell"},{"key":"4533_CR8","doi-asserted-by":"publisher","first-page":"602","DOI":"10.1038\/281602a0","volume":"281","author":"JC Chang","year":"1979","unstructured":"Chang, J. C., Temple, G. F., Trecartin, R. F. & Kan, Y. W. Suppression of the nonsense mutation in homozygous \u03b20 thalassaemia. Nature 281, 602\u2013603 (1979).","journal-title":"Nature"},{"key":"4533_CR9","doi-asserted-by":"publisher","first-page":"537","DOI":"10.1038\/296537a0","volume":"296","author":"GF Temple","year":"1982","unstructured":"Temple, G. F., Dozy, A. M., Roy, K. L. & Kan, Y. W. Construction of a functional human suppressor tRNA gene: an approach to gene therapy for \u03b2-thalassaemia. Nature 296, 537\u2013540 (1982).","journal-title":"Nature"},{"key":"4533_CR10","doi-asserted-by":"publisher","first-page":"e1641","DOI":"10.1002\/wrna.1641","volume":"12","author":"JJ Porter","year":"2021","unstructured":"Porter, J. J., Heil, C. S. & Lueck, J. D. Therapeutic promise of engineered nonsense suppressor tRNAs. Wiley Interdiscip. Rev. RNA. 12, e1641 (2021).","journal-title":"Wiley Interdiscip. Rev. RNA."},{"key":"4533_CR11","doi-asserted-by":"publisher","first-page":"62","DOI":"10.1016\/j.ymgme.2009.08.002","volume":"99","author":"D Wang","year":"2010","unstructured":"Wang, D. et al. Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation. Mol. Genet. Metab. 99, 62\u201371 (2010).","journal-title":"Mol. Genet. Metab."},{"key":"4533_CR12","doi-asserted-by":"publisher","first-page":"322","DOI":"10.1016\/j.ymgme.2018.08.003","volume":"125","author":"BW Bigger","year":"2018","unstructured":"Bigger, B. W., Begley, D. J., Virgintino, D. & Pshezhetsky, A. V. Anatomical changes and pathophysiology of the brain in mucopolysaccharidosis disorders. Mol. Genet. Metab. 125, 322\u2013331 (2018).","journal-title":"Mol. Genet. Metab."},{"key":"4533_CR13","doi-asserted-by":"publisher","first-page":"189","DOI":"10.3390\/biom11020189","volume":"11","author":"CS Hampe","year":"2021","unstructured":"Hampe, C. S. et al. Mucopolysaccharidosis type I: current treatments, limitations, and prospects for improvement. Biomolecules 11, 189 (2021).","journal-title":"Biomolecules"},{"key":"4533_CR14","doi-asserted-by":"publisher","first-page":"218","DOI":"10.1126\/science.1168978","volume":"324","author":"NT Ingolia","year":"2009","unstructured":"Ingolia, N. T., Ghaemmaghami, S., Newman, J. R. & Weissman, J. S. Genome-wide analysis in vivo of translation with nucleotide resolution using ribosome profiling. Science 324, 218\u2013223 (2009).","journal-title":"Science"},{"key":"4533_CR15","doi-asserted-by":"publisher","first-page":"e52611","DOI":"10.7554\/eLife.52611","volume":"9","author":"JR Wangen","year":"2020","unstructured":"Wangen, J. R. & Green, R. Stop codon context influences genome-wide stimulation of termination codon readthrough by aminoglycosides. eLife. 9, e52611 (2020).","journal-title":"eLife."},{"key":"4533_CR16","doi-asserted-by":"publisher","first-page":"1802","DOI":"10.1016\/j.molcel.2021.01.028","volume":"81","author":"A Behrens","year":"2021","unstructured":"Behrens, A., Rodschinka, G. & Nedialkova, D. D. High-resolution quantitative profiling of tRNA abundance and modification status in eukaryotes by mim-tRNAseq. Mol. Cell 81, 1802\u20131815.e7 (2021).","journal-title":"Mol. Cell"},{"key":"4533_CR17","doi-asserted-by":"publisher","first-page":"93","DOI":"10.1002\/jgm.2696","volume":"15","author":"R Koukuntla","year":"2013","unstructured":"Koukuntla, R., Ramsey, W. J., Young, W. B. & Link, C. J. U6 promoter-enhanced GlnUAG suppressor tRNA has higher suppression efficacy and can be stably expressed in 293 cells. J. Gene Med. 15, 93\u2013101 (2013).","journal-title":"J. Gene Med."},{"key":"4533_CR18","doi-asserted-by":"publisher","first-page":"371","DOI":"10.1146\/annurev-genom-091212-153527","volume":"15","author":"KM Keeling","year":"2014","unstructured":"Keeling, K. M., Xue, X., Gunn, G. & Bedwell, D. M. Therapeutics based on stop codon readthrough. Annu. Rev. Genomics Hum. Genet. 15, 371\u2013394 (2014).","journal-title":"Annu. Rev. Genomics Hum. Genet."},{"key":"4533_CR19","doi-asserted-by":"publisher","first-page":"1044","DOI":"10.1017\/S1355838200000716","volume":"6","author":"M Manuvakhova","year":"2000","unstructured":"Manuvakhova, M., Keeling, K. & Bedwell, D. M. Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system. RNA\u00a06, 1044\u20131055 (2000).","journal-title":"RNA"},{"key":"4533_CR20","doi-asserted-by":"publisher","first-page":"6593","DOI":"10.1128\/MCB.15.12.6593","volume":"15","author":"MK Phillips-Jones","year":"1995","unstructured":"Phillips-Jones, M. K., Hill, L. S., Atkinson, J. & Martin, R. Context effects on misreading and suppression at UAG codons in human cells. Mol. Cell. Biol. 15, 6593\u20136600 (1995).","journal-title":"Mol. Cell. Biol."},{"key":"4533_CR21","doi-asserted-by":"publisher","first-page":"12508","DOI":"10.1073\/pnas.1605336113","volume":"113","author":"B Roy","year":"2016","unstructured":"Roy, B. et al. Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression. Proc. Natl Acad. Sci. USA 113, 12508\u201312513 (2016).","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"4533_CR22","doi-asserted-by":"publisher","first-page":"3116","DOI":"10.1093\/hmg\/ddx196","volume":"26","author":"X Xue","year":"2017","unstructured":"Xue, X. et al. Identification of the amino acids inserted during suppression of CFTR nonsense mutations and determination of their functional consequences. Hum. Mol. Genet. 26, 3116\u20133129 (2017).","journal-title":"Hum. Mol. Genet."},{"key":"4533_CR23","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-019-08329-4","volume":"10","author":"JD Lueck","year":"2019","unstructured":"Lueck, J. D. et al. Engineered transfer RNAs for suppression of premature termination codons. Nat. Commun. 10, 822 (2019).","journal-title":"Nat. Commun."},{"key":"4533_CR24","doi-asserted-by":"publisher","first-page":"5017","DOI":"10.1093\/nar\/26.22.5017","volume":"26","author":"R Giege","year":"1998","unstructured":"Giege, R., Sissler, M. & Florentz, C. Universal rules and idiosyncratic features in tRNA identity. Nucleic Acids Res. 26, 5017\u20135035 (1998).","journal-title":"Nucleic Acids Res."},{"key":"4533_CR25","doi-asserted-by":"publisher","first-page":"249","DOI":"10.1016\/S0925-4439(98)00046-5","volume":"1407","author":"S Bunge","year":"1998","unstructured":"Bunge, S. et al. Genotype\u2013phenotype correlations in mucopolysaccharidosis type I using enzyme kinetics, immunoquantification and in vitro turnover studies. Biochim. Biophys. Acta 1407, 249\u2013256 (1998).","journal-title":"Biochim. Biophys. Acta"},{"key":"4533_CR26","doi-asserted-by":"publisher","first-page":"377","DOI":"10.1016\/j.ymgme.2013.05.016","volume":"109","author":"E Oussoren","year":"2013","unstructured":"Oussoren, E. et al. Residual \u03b1-l-iduronidase activity in fibroblasts of mild to severe mucopolysaccharidosis type I patients. Mol. Genet. Metab. 109, 377\u2013381 (2013).","journal-title":"Mol. Genet. Metab."},{"key":"4533_CR27","doi-asserted-by":"publisher","first-page":"121","DOI":"10.1016\/j.cels.2020.07.005","volume":"11","author":"DJ Parker","year":"2020","unstructured":"Parker, D. J. et al. Growth-optimized aminoacyl-tRNA synthetase levels prevent maximal tRNA charging. Cell Syst. 11, 121\u2013130.e6 (2020).","journal-title":"Cell Syst."},{"key":"4533_CR28","doi-asserted-by":"publisher","first-page":"407","DOI":"10.1146\/annurev.micro.59.031805.133833","volume":"59","author":"AG Hinnebusch","year":"2005","unstructured":"Hinnebusch, A. G. Translational regulation of GCN4 and the general amino acid control of yeast. Annu. Rev. Microbiol. 59, 407\u2013450 (2005).","journal-title":"Annu. Rev. Microbiol."},{"key":"4533_CR29","doi-asserted-by":"publisher","first-page":"3116","DOI":"10.1128\/MCB.20.9.3116-3124.2000","volume":"20","author":"M Buvoli","year":"2000","unstructured":"Buvoli, M., Buvoli, A. & Leinwand, L. A. Suppression of nonsense mutations in cell culture and mice by multimerized suppressor tRNA genes. Mol. Cell. Biol. 20, 3116\u20133124 (2000).","journal-title":"Mol. Cell. Biol."},{"key":"4533_CR30","doi-asserted-by":"publisher","first-page":"1363","DOI":"10.1016\/j.ymthe.2017.03.028","volume":"25","author":"J Xie","year":"2017","unstructured":"Xie, J. et al. Short DNA hairpins compromise recombinant adeno-associated virus genome homogeneity. Mol. Ther. 25, 1363\u20131374 (2017).","journal-title":"Mol. Ther."},{"key":"4533_CR31","doi-asserted-by":"publisher","first-page":"480","DOI":"10.1182\/blood-2002-09-2889","volume":"102","author":"AM Davidoff","year":"2003","unstructured":"Davidoff, A. M., Ng, C. Y., Zhou, J., Spence, Y. & Nathwani, A. C. Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood\u00a0102, 480\u2013488 (2003).","journal-title":"Blood"},{"key":"4533_CR32","doi-asserted-by":"publisher","first-page":"691","DOI":"10.1261\/rna.5147804","volume":"10","author":"KM Keeling","year":"2004","unstructured":"Keeling, K. M. et al. Leaky termination at premature stop codons antagonizes nonsense-mediated mRNA decay in S. cerevisiae. RNA 10, 691\u2013703 (2004).","journal-title":"RNA"},{"key":"4533_CR33","doi-asserted-by":"publisher","first-page":"195","DOI":"10.1016\/j.cell.2004.11.050","volume":"120","author":"YK Kim","year":"2005","unstructured":"Kim, Y. K., Furic, L., Desgroseillers, L. & Maquat, L. E. Mammalian Staufen1 recruits Upf1 to specific mRNA 3\u2032UTRs so as to elicit mRNA decay. Cell 120, 195\u2013208 (2005).","journal-title":"Cell"},{"key":"4533_CR34","doi-asserted-by":"publisher","first-page":"368","DOI":"10.1016\/j.cell.2010.07.022","volume":"142","author":"LE Maquat","year":"2010","unstructured":"Maquat, L. E., Tarn, W. Y. & Isken, O. The pioneer round of translation: features and functions. Cell 142, 368\u2013374 (2010).","journal-title":"Cell"},{"key":"4533_CR35","doi-asserted-by":"publisher","first-page":"1951","DOI":"10.1093\/nar\/16.5.1951","volume":"16","author":"H van Tol","year":"1988","unstructured":"van Tol, H. & Beier, H. All human tRNATyr genes contain introns as a prerequisite for pseudouridine biosynthesis in the anticodon. Nucleic Acids Res. 16, 1951\u20131966 (1988).","journal-title":"Nucleic Acids Res."},{"key":"4533_CR36","doi-asserted-by":"publisher","first-page":"269","DOI":"10.1016\/S1097-2765(00)00028-9","volume":"6","author":"J Dong","year":"2000","unstructured":"Dong, J., Qiu, H., Garcia-Barrio, M., Anderson, J. & Hinnebusch, A. G. Uncharged tRNA activates GCN2 by displacing the protein kinase moiety from a bipartite tRNA-binding domain. Mol. Cell 6, 269\u2013279 (2000).","journal-title":"Mol. Cell"},{"key":"4533_CR37","doi-asserted-by":"publisher","first-page":"1725","DOI":"10.1021\/bi992276t","volume":"39","author":"P Fechter","year":"2000","unstructured":"Fechter, P., Rudinger-Thirion, J., Theobald-Dietrich, A. & Giege, R. Identity of tRNA for yeast tyrosyl-tRNA synthetase: tyrosylation is more sensitive to identity nucleotides than to structural features. Biochemistry 39, 1725\u20131733 (2000).","journal-title":"Biochemistry"},{"key":"4533_CR38","doi-asserted-by":"publisher","first-page":"406","DOI":"10.1038\/s41580-019-0126-2","volume":"20","author":"T Kurosaki","year":"2019","unstructured":"Kurosaki, T., Popp, M. W. & Maquat, L. E. Quality and quantity control of gene expression by nonsense-mediated mRNA decay. Nat. Rev. Mol. Cell Biol. 20, 406\u2013420 (2019).","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"4533_CR39","doi-asserted-by":"publisher","first-page":"189","DOI":"10.1261\/rna.059055.116","volume":"23","author":"M Colombo","year":"2017","unstructured":"Colombo, M., Karousis, E. D., Bourquin, J., Bruggmann, R. & Muhlemann, O. Transcriptome-wide identification of NMD-targeted human mRNAs reveals extensive redundancy between SMG6- and SMG7-mediated degradation pathways. RNA 23, 189\u2013201 (2017).","journal-title":"RNA"},{"key":"4533_CR40","doi-asserted-by":"publisher","first-page":"175","DOI":"10.1089\/nat.2019.0779","volume":"29","author":"L Huang","year":"2019","unstructured":"Huang, L. et al. Targeting translation termination machinery with antisense oligonucleotides for diseases caused by nonsense mutations. Nucleic Acid Ther. 29, 175\u2013186 (2019).","journal-title":"Nucleic Acid Ther."},{"key":"4533_CR41","first-page":"S128","volume":"29","author":"J Wang","year":"2021","unstructured":"Wang, J. et al. In vivo delivery of suppressor tRNA overcomes a pathogenic nonsense mutation in mice. Mol. Ther. 29, S128 (2021).","journal-title":"Mol. Ther."},{"key":"4533_CR42","doi-asserted-by":"publisher","first-page":"1172","DOI":"10.1038\/nn.4593","volume":"20","author":"KY Chan","year":"2017","unstructured":"Chan, K. Y. et al. Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems. Nat. Neurosci. 20, 1172\u20131179 (2017).","journal-title":"Nat. Neurosci."},{"key":"4533_CR43","doi-asserted-by":"publisher","first-page":"806","DOI":"10.1038\/s41551-019-0431-2","volume":"3","author":"AM Moreno","year":"2019","unstructured":"Moreno, A. M. et al. Immune-orthogonal orthologues of AAV capsids and of Cas9 circumvent the immune response to the administration of gene therapy. Nat. Biomed. Eng. 3, 806\u2013816 (2019).","journal-title":"Nat. Biomed. Eng."},{"key":"4533_CR44","doi-asserted-by":"publisher","first-page":"1432","DOI":"10.1016\/j.ymthe.2020.04.017","volume":"28","author":"A Li","year":"2020","unstructured":"Li, A. et al. AAV-CRISPR gene editing is negated by pre-existing immunity to Cas9. Mol. Ther. 28, 1432\u20131441 (2020).","journal-title":"Mol. Ther."},{"key":"4533_CR45","doi-asserted-by":"publisher","first-page":"18","DOI":"10.1038\/mt.2012.200","volume":"21","author":"KK Gadalla","year":"2013","unstructured":"Gadalla, K. K. et al. Improved survival and reduced phenotypic severity following AAV9\/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice. Mol. Ther. 21, 18\u201330 (2013).","journal-title":"Mol. Ther."},{"key":"4533_CR46","doi-asserted-by":"publisher","first-page":"3747","DOI":"10.1093\/nar\/gkaa136","volume":"48","author":"L Kramarski","year":"2020","unstructured":"Kramarski, L. & Arbely, E. Translational read-through promotes aggregation and shapes stop codon identity. Nucleic Acids Res. 48, 3747\u20133760 (2020).","journal-title":"Nucleic Acids Res."},{"key":"4533_CR47","doi-asserted-by":"publisher","first-page":"777","DOI":"10.1002\/1873-3468.13362","volume":"593","author":"S Hashimoto","year":"2019","unstructured":"Hashimoto, S., Nobuta, R., Izawa, T. & Inada, T. Translation arrest as a protein quality control system for aberrant translation of the 3\u2032-UTR in mammalian cells. FEBS Lett. 593, 777\u2013787 (2019).","journal-title":"FEBS Lett."},{"key":"4533_CR48","doi-asserted-by":"publisher","first-page":"719","DOI":"10.1038\/nature18308","volume":"534","author":"JA Arribere","year":"2016","unstructured":"Arribere, J. A. et al. Translation readthrough mitigation. Nature 534, 719\u2013723 (2016).","journal-title":"Nature"},{"key":"4533_CR49","doi-asserted-by":"publisher","first-page":"254","DOI":"10.1080\/15476286.2019.1676115","volume":"17","author":"S Lombardi","year":"2020","unstructured":"Lombardi, S. et al. Translational readthrough of GLA nonsense mutations suggests dominant-negative effects exerted by the interaction of wild-type and missense variants. RNA Biol. 17, 254\u2013263 (2020).","journal-title":"RNA Biol."},{"key":"4533_CR50","doi-asserted-by":"publisher","first-page":"173","DOI":"10.1038\/d41573-021-00017-7","volume":"20","author":"DA Kuzmin","year":"2021","unstructured":"Kuzmin, D. A. et al. The clinical landscape for AAV gene therapies. Nat. Rev. Drug Discov. 20, 173\u2013174 (2021).","journal-title":"Nat. Rev. Drug Discov."},{"key":"4533_CR51","doi-asserted-by":"publisher","first-page":"5383","DOI":"10.1021\/ac025747h","volume":"74","author":"A Keller","year":"2002","unstructured":"Keller, A., Nesvizhskii, A. I., Kolker, E. & Aebersold, R. Empirical statistical model to estimate the accuracy of peptide identifications made by MS\/MS and database search. Anal. Chem. 74, 5383\u20135392 (2002).","journal-title":"Anal. Chem."},{"key":"4533_CR52","doi-asserted-by":"publisher","first-page":"4646","DOI":"10.1021\/ac0341261","volume":"75","author":"AI Nesvizhskii","year":"2003","unstructured":"Nesvizhskii, A. I., Keller, A., Kolker, E. & Aebersold, R. A statistical model for identifying proteins by tandem mass spectrometry. Anal. Chem. 75, 4646\u20134658 (2003).","journal-title":"Anal. Chem."},{"key":"4533_CR53","doi-asserted-by":"publisher","first-page":"1534","DOI":"10.1038\/nprot.2012.086","volume":"7","author":"NT Ingolia","year":"2012","unstructured":"Ingolia, N. T., Brar, G. A., Rouskin, S., McGeachy, A. M. & Weissman, J. S. The ribosome profiling strategy for monitoring translation in vivo by deep sequencing of ribosome-protected mRNA fragments. Nat. Protoc. 7, 1534\u20131550 (2012).","journal-title":"Nat. Protoc."},{"key":"4533_CR54","doi-asserted-by":"publisher","first-page":"15","DOI":"10.1093\/bioinformatics\/bts635","volume":"29","author":"A Dobin","year":"2013","unstructured":"Dobin, A. et al. STAR: ultrafast universal RNA-seq aligner. Bioinformatics 29, 15\u201321 (2013).","journal-title":"Bioinformatics"},{"key":"4533_CR55","doi-asserted-by":"publisher","DOI":"10.1186\/s12864-020-6714-x","volume":"21","author":"O Yukselen","year":"2020","unstructured":"Yukselen, O., Turkyilmaz, O., Ozturk, A. R., Garber, M. & Kucukural, A. DolphinNext: a distributed data processing platform for high throughput genomics. BMC Genomics 21, 310 (2020).","journal-title":"BMC Genomics"},{"key":"4533_CR56","doi-asserted-by":"publisher","first-page":"503","DOI":"10.1093\/hmg\/6.4.503","volume":"6","author":"LA Clarke","year":"1997","unstructured":"Clarke, L. A. et al. Murine mucopolysaccharidosis type I: targeted disruption of the murine \u03b1-l-iduronidase gene. Hum. Mol. Genet. 6, 503\u2013511 (1997).","journal-title":"Hum. Mol. Genet."},{"key":"4533_CR57","doi-asserted-by":"publisher","first-page":"116","DOI":"10.1016\/j.ymgme.2011.10.005","volume":"105","author":"D Wang","year":"2012","unstructured":"Wang, D. et al. The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse. Mol. Genet. Metab. 105, 116\u2013125 (2012).","journal-title":"Mol. Genet. Metab."},{"key":"4533_CR58","doi-asserted-by":"publisher","first-page":"839","DOI":"10.1038\/nbt.4219","volume":"36","author":"D Wang","year":"2018","unstructured":"Wang, D. et al. Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice. Nat. Biotechnol. 36, 839\u2013842 (2018).","journal-title":"Nat. Biotechnol."},{"key":"4533_CR59","first-page":"e3465","volume":"9","author":"AR Crowe","year":"2019","unstructured":"Crowe, A. R. & Yue, W. Semi-quantitative determination of protein expression using immunohistochemistry staining and analysis: an integrated protocol. Bio Protoc. 9, e3465 (2019).","journal-title":"Bio Protoc."},{"key":"4533_CR60","doi-asserted-by":"publisher","first-page":"2114","DOI":"10.1093\/bioinformatics\/btu170","volume":"30","author":"AM Bolger","year":"2014","unstructured":"Bolger, A. M., Lohse, M. & Usadel, B. Trimmomatic: a flexible trimmer for Illumina sequence data. Bioinformatics 30, 2114\u20132120 (2014).","journal-title":"Bioinformatics"},{"key":"4533_CR61","doi-asserted-by":"publisher","first-page":"e133","DOI":"10.1093\/nar\/gkx514","volume":"45","author":"ME Evans","year":"2017","unstructured":"Evans, M. E., Clark, W. C., Zheng, G. & Pan, T. Determination of tRNA aminoacylation levels by high-throughput sequencing. Nucleic Acids Res. 45, e133 (2017).","journal-title":"Nucleic Acids Res."}],"container-title":["Nature"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.nature.com\/articles\/s41586-022-04533-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41586-022-04533-3","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41586-022-04533-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,5,12]],"date-time":"2022-05-12T16:15:08Z","timestamp":1652372108000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/s41586-022-04533-3"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,3,23]]},"references-count":61,"journal-issue":{"issue":"7905","published-print":{"date-parts":[[2022,4,14]]}},"alternative-id":["4533"],"URL":"https:\/\/doi.org\/10.1038\/s41586-022-04533-3","relation":{},"ISSN":["0028-0836","1476-4687"],"issn-type":[{"value":"0028-0836","type":"print"},{"value":"1476-4687","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,3,23]]},"assertion":[{"value":"9 July 2021","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"5 January 2022","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"23 March 2022","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"12 May 2022","order":4,"name":"change_date","label":"Change Date","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Update","order":5,"name":"change_type","label":"Change Type","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"In the version of this article initially published, the name of John Leuck was not included in the Peer review information for this article, which has now been amended.","order":6,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"J.W., Y.Z., C.A.M., G.G. and D.W. are inventors of a patent application filed by the University of Massachusetts Chan Medical School concerning the design and applications of sup-tRNAs described in this study. G.G. is a scientific co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics and holds equity in these companies. G.G. and D.W. are inventors on patents related to AAV-based gene therapy, some of which were licensed to commercial entities. The other authors declare no competing interests.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}]}}