{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,17]],"date-time":"2026-03-17T21:04:11Z","timestamp":1773781451063,"version":"3.50.1"},"reference-count":91,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2025,3,1]],"date-time":"2025-03-01T00:00:00Z","timestamp":1740787200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,3,1]],"date-time":"2025-03-01T00:00:00Z","timestamp":1740787200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,12,19]],"date-time":"2024-12-19T00:00:00Z","timestamp":1734566400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000024","name":"Canadian Institutes of Health Research","doi-asserted-by":"publisher","award":["165985"],"award-info":[{"award-number":["165985"]}],"id":[{"id":"10.13039\/501100000024","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100008093","name":"Western University Department of Family Medicine","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100008093","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100009836","name":"Huntington Society of Canada","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100009836","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000038","name":"Natural Sciences and Engineering Research Council of Canada","doi-asserted-by":"publisher","award":["580241"],"award-info":[{"award-number":["580241"]}],"id":[{"id":"10.13039\/501100000038","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000038","name":"Natural Sciences and Engineering Research Council of Canada","doi-asserted-by":"publisher","award":["04282"],"award-info":[{"award-number":["04282"]}],"id":[{"id":"10.13039\/501100000038","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001804","name":"Canada Research Chairs Program","doi-asserted-by":"publisher","award":["232341"],"award-info":[{"award-number":["232341"]}],"id":[{"id":"10.13039\/501100001804","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004381","name":"Western University","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004381","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Nucleic Acids"],"published-print":{"date-parts":[[2025,3]]},"DOI":"10.1016\/j.omtn.2024.102442","type":"journal-article","created":{"date-parts":[[2024,12,21]],"date-time":"2024-12-21T11:35:25Z","timestamp":1734780925000},"page":"102442","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":4,"title":["Glutamine missense suppressor transfer RNAs inhibit polyglutamine aggregation"],"prefix":"10.1016","volume":"36","author":[{"given":"Rasangi","family":"Tennakoon","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Teija M.I.","family":"Bily","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Farah","family":"Hasan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sunidhi","family":"Syal","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Aaron","family":"Voigt","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tugce B.","family":"Balci","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kyle S.","family":"Hoffman","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Patrick","family":"O\u2019Donoghue","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2024.102442_bib1","doi-asserted-by":"crossref","first-page":"575","DOI":"10.1146\/annurev.neuro.29.051605.113042","article-title":"Trinucleotide repeat disorders","volume":"30","author":"Orr","year":"2007","journal-title":"Annu. Rev. Neurosci."},{"key":"10.1016\/j.omtn.2024.102442_bib2","doi-asserted-by":"crossref","first-page":"1401","DOI":"10.1056\/NEJM199405193302001","article-title":"A worldwide study of the Huntington's disease mutation. The sensitivity and specificity of measuring CAG repeats","volume":"330","author":"Kremer","year":"1994","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtn.2024.102442_bib3","doi-asserted-by":"crossref","first-page":"887","DOI":"10.1016\/j.cell.2019.06.036","article-title":"CAG Repeat Not Polyglutamine Length Determines Timing of Huntington's Disease Onset","volume":"178","year":"2019","journal-title":"Cell"},{"key":"10.1016\/j.omtn.2024.102442_bib4","doi-asserted-by":"crossref","first-page":"172","DOI":"10.1016\/j.ajhg.2021.12.002","article-title":"Longer CAG repeat length is associated with shorter survival after disease onset in Huntington disease","volume":"109","author":"Langbehn","year":"2022","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.omtn.2024.102442_bib5","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2020.104963","article-title":"The contribution of glial cells to Huntington's disease pathogenesis","volume":"143","author":"Wilton","year":"2020","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtn.2024.102442_bib6","first-page":"65","article-title":"The biological function of the Huntingtin protein and its relevance to Huntington's Disease pathology","volume":"5","author":"Schulte","year":"2011","journal-title":"Curr. Trends Neurol."},{"key":"10.1016\/j.omtn.2024.102442_bib7","doi-asserted-by":"crossref","first-page":"8820","DOI":"10.1021\/jacs.7b00838","article-title":"Emerging \u03b2-Sheet Rich Conformations in Supercompact Huntingtin Exon-1 Mutant Structures","volume":"139","author":"Kang","year":"2017","journal-title":"J.\u00a0Am. Chem. Soc."},{"key":"10.1016\/j.omtn.2024.102442_bib8","doi-asserted-by":"crossref","first-page":"1052","DOI":"10.1111\/jnc.13463","article-title":"Glyceraldehyde 3-phosphate dehydrogenase augments the intercellular transmission and toxicity of polyglutamine aggregates in a cell model of Huntington disease","volume":"136","author":"Mikhaylova","year":"2016","journal-title":"J.\u00a0Neurochem."},{"key":"10.1016\/j.omtn.2024.102442_bib9","doi-asserted-by":"crossref","first-page":"325","DOI":"10.1016\/B978-0-12-381328-2.00014-6","article-title":"Genetics and neuropathology of Huntington's disease","volume":"98","author":"Reiner","year":"2011","journal-title":"Int. Rev. Neurobiol."},{"key":"10.1016\/j.omtn.2024.102442_bib10","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1007\/s40259-022-00519-9","article-title":"Antisense Oligonucleotide Therapy: From Design to the Huntington Disease Clinic","volume":"36","author":"Rook","year":"2022","journal-title":"BioDrugs"},{"key":"10.1016\/j.omtn.2024.102442_bib11","doi-asserted-by":"crossref","first-page":"2307","DOI":"10.1056\/NEJMoa1900907","article-title":"Targeting Huntingtin Expression in Patients with Huntington's Disease","volume":"380","author":"Tabrizi","year":"2019","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtn.2024.102442_bib12","doi-asserted-by":"crossref","first-page":"1","DOI":"10.3233\/JHD-240017","article-title":"Huntington's Disease Clinical Trials Corner: March 2024","volume":"13","author":"Estevez-Fraga","year":"2024","journal-title":"J.\u00a0Huntingtons Dis."},{"key":"10.1016\/j.omtn.2024.102442_bib13","doi-asserted-by":"crossref","first-page":"e297","DOI":"10.1038\/mtna.2016.7","article-title":"Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease","volume":"5","author":"Miniarikova","year":"2016","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2024.102442_bib14","doi-asserted-by":"crossref","first-page":"102","DOI":"10.1016\/j.omtn.2022.06.004","article-title":"A peptide inhibitor that rescues polyglutamine-induced synaptic defects and cell death through suppressing RNA and protein toxicities","volume":"29","author":"Peng","year":"2022","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2024.102442_bib15","doi-asserted-by":"crossref","first-page":"505","DOI":"10.1016\/j.jmb.2011.07.003","article-title":"Aggregation kinetics of interrupted polyglutamine peptides","volume":"412","author":"Walters","year":"2011","journal-title":"J.\u00a0Mol. Biol."},{"key":"10.1016\/j.omtn.2024.102442_bib16","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1096\/fj.03-1238fje","article-title":"Inhibition of polyglutamine aggregate cytotoxicity by a structure-based elongation inhibitor","volume":"18","author":"Thakur","year":"2004","journal-title":"Faseb. J."},{"key":"10.1016\/j.omtn.2024.102442_bib17","doi-asserted-by":"crossref","first-page":"469","DOI":"10.1093\/protein\/gzp023","article-title":"The impact of ataxin-1-like histidine insertions on polyglutamine aggregation","volume":"22","author":"Jayaraman","year":"2009","journal-title":"Protein Eng. Des. Sel."},{"key":"10.1016\/j.omtn.2024.102442_bib18","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1016\/j.celrep.2012.12.019","article-title":"Depletion of cognate charged transfer RNA causes translational frameshifting within the expanded CAG stretch in huntingtin","volume":"3","author":"Girstmair","year":"2013","journal-title":"Cell Rep."},{"key":"10.1016\/j.omtn.2024.102442_bib19","doi-asserted-by":"crossref","first-page":"18505","DOI":"10.1074\/jbc.M116.744326","article-title":"An Expanded CAG Repeat in Huntingtin Causes\u00a0+1 Frameshifting","volume":"291","author":"Saffert","year":"2016","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtn.2024.102442_bib20","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1016\/j.csbj.2021.12.029","article-title":"Small-molecule modulators of TRMT2A decrease PolyQ aggregation and PolyQ-induced cell death","volume":"20","author":"Margreiter","year":"2022","journal-title":"Comput. Struct. Biotechnol. J."},{"key":"10.1016\/j.omtn.2024.102442_bib21","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0047452","article-title":"Large-scale screen for modifiers of ataxin-3-derived polyglutamine-induced toxicity in Drosophila","volume":"7","author":"VoSSfeldt","year":"2012","journal-title":"PLoS One"},{"key":"10.1016\/j.omtn.2024.102442_bib22","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.161769","article-title":"Benefits of global mutant huntingtin lowering diminish over time in a Huntington's disease mouse model","volume":"7","author":"Marchionini","year":"2022","journal-title":"JCI Insight"},{"key":"10.1016\/j.omtn.2024.102442_bib23","doi-asserted-by":"crossref","first-page":"801","DOI":"10.1016\/j.neuron.2019.01.039","article-title":"Huntingtin Lowering Strategies for Disease Modification in Huntington's Disease","volume":"101","author":"Tabrizi","year":"2019","journal-title":"Neuron"},{"key":"10.1016\/j.omtn.2024.102442_bib24","doi-asserted-by":"crossref","first-page":"550","DOI":"10.1128\/MMBR.67.4.550-573.2003","article-title":"On the evolution of structure in aminoacyl-tRNA synthetases","volume":"67","author":"O'Donoghue","year":"2003","journal-title":"Microbiol. Mol. Biol. Rev."},{"key":"10.1016\/j.omtn.2024.102442_bib25","doi-asserted-by":"crossref","first-page":"5017","DOI":"10.1093\/nar\/26.22.5017","article-title":"Universal rules and idiosyncratic features in tRNA identity","volume":"26","author":"Gieg\u00e9","year":"1998","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2024.102442_bib26","doi-asserted-by":"crossref","first-page":"1528","DOI":"10.1093\/nar\/gkad007","article-title":"The tRNA identity landscape for aminoacylation and beyond","volume":"51","author":"Gieg\u00e9","year":"2023","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2024.102442_bib27","doi-asserted-by":"crossref","first-page":"11883","DOI":"10.1093\/nar\/gkab898","article-title":"Formation and persistence of polyglutamine aggregates in mistranslating cells","volume":"49","author":"Lant","year":"2021","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2024.102442_bib28","doi-asserted-by":"crossref","DOI":"10.1098\/rstb.2022.0029","article-title":"Perseverance of protein homeostasis despite mistranslation of glycine codons with alanine","volume":"378","author":"Hasan","year":"2023","journal-title":"Philos. Trans. R. Soc. Lond. B Biol. Sci."},{"key":"10.1016\/j.omtn.2024.102442_bib29","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1080\/15476286.2024.2340297","article-title":"Mistranslating the genetic code with leucine in yeast and mammalian cells","volume":"21","author":"Davey-Young","year":"2024","journal-title":"RNA Biol."},{"key":"10.1016\/j.omtn.2024.102442_bib30","doi-asserted-by":"crossref","first-page":"5294","DOI":"10.1074\/jbc.REV118.002982","article-title":"Pathways to disease from natural variations in human cytoplasmic tRNAs","volume":"294","author":"Lant","year":"2019","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtn.2024.102442_bib31","doi-asserted-by":"crossref","DOI":"10.3390\/genes14020518","article-title":"Genetic Interaction of tRNA-Dependent Mistranslation with Fused in Sarcoma Protein Aggregates","volume":"14","author":"Lant","year":"2023","journal-title":"Genes"},{"key":"10.1016\/j.omtn.2024.102442_bib32","doi-asserted-by":"crossref","first-page":"273","DOI":"10.1016\/j.ymthe.2024.01.001","article-title":"Missense suppressor tRNA therapeutics correct disease-causing alleles by misreading the genetic code","volume":"32","author":"Bily","year":"2024","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2024.102442_bib33","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1016\/j.ymthe.2023.12.014","article-title":"Engineered mischarged transfer RNAs for correcting pathogenic missense mutations","volume":"32","author":"Hou","year":"2024","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2024.102442_bib34","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."},{"key":"10.1016\/j.omtn.2024.102442_bib35","doi-asserted-by":"crossref","first-page":"7976","DOI":"10.1021\/acs.chemrev.4c00142","article-title":"Mechanisms and Delivery of tRNA Therapeutics","volume":"124","author":"Ward","year":"2024","journal-title":"Chem. Rev."},{"key":"10.1016\/j.omtn.2024.102442_bib36","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1016\/j.bbrc.2012.09.028","article-title":"Anti-tumor effects of an engineered \"killer\" transfer RNA","volume":"427","author":"Zhou","year":"2012","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.omtn.2024.102442_bib37","doi-asserted-by":"crossref","first-page":"2635","DOI":"10.1016\/S0021-9258(19)69661-4","article-title":"Codon reading and translational error. Reading of the glutamine and lysine codons during protein synthesis in\u00a0vitro","volume":"256","author":"Lustig","year":"1981","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtn.2024.102442_bib38","doi-asserted-by":"crossref","first-page":"D184","DOI":"10.1093\/nar\/gkv1309","article-title":"GtRNAdb 2.0: an expanded database of transfer RNA genes identified in complete and draft genomes","volume":"44","author":"Chan","year":"2016","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2024.102442_bib39","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1007\/978-1-4939-6807-7_15","article-title":"High-Throughput Small RNA Sequencing Enhanced by AlkB-Facilitated RNA de-Methylation (ARM-Seq)","volume":"1562","author":"Hrabeta-Robinson","year":"2017","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.omtn.2024.102442_bib40","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1038\/nature11247","article-title":"An integrated encyclopedia of DNA elements in the human genome","volume":"489","year":"2012","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2024.102442_bib89","doi-asserted-by":"crossref","first-page":"1853","DOI":"10.4161\/rna.27361","article-title":"Diversity of human tRNA genes from the 1000-genomes project","volume":"10","author":"Parisien","year":"2013","journal-title":"RNA Biol."},{"key":"10.1016\/j.omtn.2024.102442_bib90","doi-asserted-by":"crossref","first-page":"D853","DOI":"10.1093\/nar\/gky1095","article-title":"The UCSC Genome Browser database: 2019 update","volume":"47","author":"Haeussler","year":"2019","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2024.102442_bib91","doi-asserted-by":"crossref","first-page":"580","DOI":"10.1038\/ng.2653","article-title":"The Genotype-Tissue Expression (GTEx) project","volume":"45","year":"2013","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omtn.2024.102442_bib41","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2020.104951","article-title":"Use of adeno-associated virus-mediated delivery of mutant huntingtin to study the spreading capacity of the protein in mice and non-human primates","volume":"141","author":"Maxan","year":"2020","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtn.2024.102442_bib42","doi-asserted-by":"crossref","first-page":"5896","DOI":"10.1038\/sj.emboj.7601445","article-title":"Huntingtin inhibits caspase-3 activation","volume":"25","author":"Zhang","year":"2006","journal-title":"EMBO J."},{"key":"10.1016\/j.omtn.2024.102442_bib43","doi-asserted-by":"crossref","first-page":"e382","DOI":"10.1038\/cddis.2012.121","article-title":"An in\u00a0vitro perspective on the molecular mechanisms underlying mutant huntingtin protein toxicity","volume":"3","author":"Cisbani","year":"2012","journal-title":"Cell Death Dis."},{"key":"10.1016\/j.omtn.2024.102442_bib44","doi-asserted-by":"crossref","DOI":"10.1093\/nar\/gkp1083","article-title":"Chimeric tRNAs as tools to induce proteome damage and identify components of stress responses","volume":"38","author":"Geslain","year":"2010","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2024.102442_bib45","doi-asserted-by":"crossref","first-page":"275","DOI":"10.1038\/nmeth.1314","article-title":"SUnSET, a nonradioactive method to monitor protein synthesis","volume":"6","author":"Schmidt","year":"2009","journal-title":"Nat. Methods"},{"key":"10.1016\/j.omtn.2024.102442_bib46","article-title":"Serine mistranslation induces the integrated stress response without accumulation of uncharged tRNAs","author":"Zhang","year":"2024","journal-title":"bioRxiv"},{"key":"10.1016\/j.omtn.2024.102442_bib47","doi-asserted-by":"crossref","first-page":"1059","DOI":"10.1042\/BCJ20220068","article-title":"The role of eIF2 phosphorylation in cell and organismal physiology: new roles for well-known actors","volume":"479","author":"Wang","year":"2022","journal-title":"Biochem. J."},{"key":"10.1016\/j.omtn.2024.102442_bib48","doi-asserted-by":"crossref","first-page":"17672","DOI":"10.1074\/jbc.RA120.015398","article-title":"AggreCount: an unbiased image analysis tool for identifying and quantifying cellular aggregates in a spatially defined manner","volume":"295","author":"Klickstein","year":"2020","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtn.2024.102442_bib49","doi-asserted-by":"crossref","first-page":"79","DOI":"10.2174\/1875397301206010079","article-title":"High-Throughput Multiplexed Quantitation of Protein Aggregation and Cytotoxicity in a Huntington's Disease Model","volume":"6","author":"Titus","year":"2012","journal-title":"Curr. Chem. Genom."},{"key":"10.1016\/j.omtn.2024.102442_bib50","doi-asserted-by":"crossref","first-page":"951","DOI":"10.1016\/j.molcel.2016.07.022","article-title":"Soluble Oligomers of PolyQ-Expanded Huntingtin Target a Multiplicity of Key Cellular Factors","volume":"63","author":"Kim","year":"2016","journal-title":"Mol. Cell"},{"key":"10.1016\/j.omtn.2024.102442_bib51","doi-asserted-by":"crossref","first-page":"8996","DOI":"10.1073\/pnas.1801240115","article-title":"Transfer RNA genes experience exceptionally elevated mutation rates","volume":"115","author":"Thornlow","year":"2018","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2024.102442_bib52","doi-asserted-by":"crossref","first-page":"459","DOI":"10.1089\/dna.1.1988.7.459","article-title":"Modulation of the phenotypic expression of a human serine tRNA gene by 5'-flanking sequences","volume":"7","author":"Capone","year":"1988","journal-title":"DNA"},{"key":"10.1016\/j.omtn.2024.102442_bib53","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1016\/S0896-6273(02)00872-3","article-title":"Polyglutamine pathogenesis: emergence of unifying mechanisms for Huntington's disease and related disorders","volume":"35","author":"Ross","year":"2002","journal-title":"Neuron"},{"key":"10.1016\/j.omtn.2024.102442_bib54","doi-asserted-by":"crossref","first-page":"8473","DOI":"10.1523\/JNEUROSCI.21-21-08473.2001","article-title":"Huntingtin aggregate-associated axonal degeneration is an early pathological event in Huntington's disease\u00a0mice","volume":"21","author":"Li","year":"2001","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.omtn.2024.102442_bib55","doi-asserted-by":"crossref","first-page":"3877","DOI":"10.1523\/JNEUROSCI.11-12-03877.1991","article-title":"Proliferative and degenerative changes in striatal spiny neurons in Huntington's disease: a combined study using the section-Golgi method and calbindin D28k immunocytochemistry","volume":"11","author":"Ferrante","year":"1991","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.omtn.2024.102442_bib56","doi-asserted-by":"crossref","first-page":"2088","DOI":"10.1212\/WNL.43.10.2088","article-title":"Evidence for neuronal degeneration and dendritic plasticity in cortical pyramidal neurons of Huntington's disease: a quantitative Golgi study","volume":"43","author":"Sotrel","year":"1993","journal-title":"Neurology"},{"key":"10.1016\/j.omtn.2024.102442_bib57","doi-asserted-by":"crossref","first-page":"2522","DOI":"10.1523\/JNEUROSCI.19-07-02522.1999","article-title":"Nuclear and neuropil aggregates in Huntington's disease: relationship to neuropathology","volume":"19","author":"Gutekunst","year":"1999","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.omtn.2024.102442_bib58","doi-asserted-by":"crossref","first-page":"581","DOI":"10.1534\/genetics.111.133710","article-title":"Huntingtin aggregation kinetics and their pathological role in a Drosophila Huntington's disease model","volume":"190","author":"Weiss","year":"2012","journal-title":"Genetics"},{"key":"10.1016\/j.omtn.2024.102442_bib59","doi-asserted-by":"crossref","first-page":"11884","DOI":"10.1073\/pnas.182276099","article-title":"Huntington's disease age-of-onset linked to polyglutamine aggregation nucleation","volume":"99","author":"Chen","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2024.102442_bib60","doi-asserted-by":"crossref","DOI":"10.1186\/1750-1326-7-20","article-title":"Quantitative connection between polyglutamine aggregation kinetics and neurodegenerative process in patients with Huntington's disease","volume":"7","author":"Sugaya","year":"2012","journal-title":"Mol. Neurodegener."},{"key":"10.1016\/j.omtn.2024.102442_bib61","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pgen.1000838","article-title":"Deletion of the huntingtin polyglutamine stretch enhances neuronal autophagy and longevity in mice","volume":"6","author":"Zheng","year":"2010","journal-title":"PLoS Genet."},{"key":"10.1016\/j.omtn.2024.102442_bib62","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1038\/ng0896-442","article-title":"Cleavage of huntingtin by apopain, a proapoptotic cysteine protease, is modulated by the polyglutamine tract","volume":"13","author":"Goldberg","year":"1996","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omtn.2024.102442_bib63","doi-asserted-by":"crossref","first-page":"9158","DOI":"10.1074\/jbc.273.15.9158","article-title":"Caspase cleavage of gene products associated with triplet expansion disorders generates truncated fragments containing the polyglutamine tract","volume":"273","author":"Wellington","year":"1998","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtn.2024.102442_bib64","doi-asserted-by":"crossref","first-page":"769","DOI":"10.1242\/dev.114.3.769","article-title":"Early mRNAs, spatially restricted along the animal-vegetal axis of sea urchin embryos, include one encoding a protein related to tolloid and BMP-1","volume":"114","author":"Reynolds","year":"1992","journal-title":"Development"},{"key":"10.1016\/j.omtn.2024.102442_bib65","doi-asserted-by":"crossref","first-page":"8808","DOI":"10.1074\/jbc.M109.075028","article-title":"Proteolysis of mutant huntingtin produces an exon 1 fragment that accumulates as an aggregated protein in neuronal nuclei in Huntington disease","volume":"285","author":"Landles","year":"2010","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtn.2024.102442_bib66","doi-asserted-by":"crossref","first-page":"2366","DOI":"10.1073\/pnas.1221891110","article-title":"Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease","volume":"110","author":"Sathasivam","year":"2013","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2024.102442_bib67","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1016\/S0092-8674(00)81369-0","article-title":"Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice","volume":"87","author":"Mangiarini","year":"1996","journal-title":"Cell"},{"key":"10.1016\/j.omtn.2024.102442_bib68","doi-asserted-by":"crossref","first-page":"4406","DOI":"10.1073\/pnas.1702237114","article-title":"Aggregation landscapes of Huntingtin exon 1 protein fragments and the critical repeat length for the onset of Huntington's disease","volume":"114","author":"Chen","year":"2017","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2024.102442_bib69","doi-asserted-by":"crossref","first-page":"D303","DOI":"10.1093\/nar\/gkx1030","article-title":"MODOMICS: a database of RNA modification pathways. 2017 update","volume":"46","author":"Boccaletto","year":"2018","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2024.102442_bib70","doi-asserted-by":"crossref","DOI":"10.3389\/fmolb.2021.769184","article-title":"Molecular Strategies to Target Protein Aggregation in Huntington's Disease","volume":"8","author":"Jarosi\u0144ska","year":"2021","journal-title":"Front. Mol. Biosci."},{"key":"10.1016\/j.omtn.2024.102442_bib71","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1016\/j.nbd.2018.08.019","article-title":"Mutational analysis implicates the amyloid fibril as the toxic entity in Huntington's disease","volume":"120","author":"Drombosky","year":"2018","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtn.2024.102442_bib72","doi-asserted-by":"crossref","DOI":"10.1038\/srep34916","article-title":"Multiple discrete soluble aggregates influence polyglutamine toxicity in a Huntington's disease model system","volume":"6","author":"Xi","year":"2016","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtn.2024.102442_bib73","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1016\/j.celrep.2017.04.029","article-title":"Huntingtin Inclusions Trigger Cellular Quiescence, Deactivate Apoptosis, and Lead to Delayed Necrosis","volume":"19","author":"Ramdzan","year":"2017","journal-title":"Cell Rep."},{"key":"10.1016\/j.omtn.2024.102442_bib74","doi-asserted-by":"crossref","first-page":"2753","DOI":"10.1038\/ncomms3753","article-title":"Soluble forms of polyQ-expanded huntingtin rather than large aggregates cause endoplasmic reticulum stress","volume":"4","author":"Leitman","year":"2013","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtn.2024.102442_bib75","doi-asserted-by":"crossref","first-page":"528","DOI":"10.1002\/jgm.378","article-title":"Antisense downregulation of mutant huntingtin in a cell model","volume":"5","author":"Hasholt","year":"2003","journal-title":"J.\u00a0Gene Med."},{"key":"10.1016\/j.omtn.2024.102442_bib76","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0024308","article-title":"Targeting several CAG expansion diseases by a single antisense oligonucleotide","volume":"6","author":"Evers","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.omtn.2024.102442_bib77","doi-asserted-by":"crossref","first-page":"3421","DOI":"10.1093\/brain\/awab354","article-title":"Therapeutic reversal of Huntington's disease by in\u00a0vivo self-assembled siRNAs","volume":"144","author":"Zhang","year":"2021","journal-title":"Brain"},{"key":"10.1016\/j.omtn.2024.102442_bib78","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1038\/s41587-022-01252-y","article-title":"tRNA therapeutics burst onto startup scene","volume":"40","author":"Dolgin","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2024.102442_bib79","doi-asserted-by":"crossref","DOI":"10.1016\/j.jbc.2023.105246","article-title":"Ushering in the era of tRNA medicines","volume":"299","author":"Anastassiadis","year":"2023","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtn.2024.102442_bib80","doi-asserted-by":"crossref","first-page":"108","DOI":"10.1038\/s41573-023-00829-9","article-title":"tRNA therapeutics for genetic diseases","volume":"23","author":"Coller","year":"2024","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omtn.2024.102442_bib81","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"},{"key":"10.1016\/j.omtn.2024.102442_bib82","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1001\/jamaneurol.2021.1272","article-title":"Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy","volume":"78","author":"Mendell","year":"2021","journal-title":"JAMA Neurol."},{"key":"10.1016\/j.omtn.2024.102442_bib83","doi-asserted-by":"crossref","first-page":"3038","DOI":"10.1182\/blood-2011-09-382317","article-title":"Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer","volume":"119","author":"Buchlis","year":"2012","journal-title":"Blood"},{"key":"10.1016\/j.omtn.2024.102442_bib84","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1089\/humc.2017.010","article-title":"Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease","volume":"28","author":"Sehara","year":"2017","journal-title":"Hum. Gene Ther. Clin. Dev."},{"key":"10.1016\/j.omtn.2024.102442_bib85","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1016\/j.omtm.2020.10.011","article-title":"CNS Transduction Benefits of AAV-PHP.eB over AAV9 Are Dependent on Administration Route and Mouse Strain","volume":"19","author":"Mathiesen","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtn.2024.102442_bib86","doi-asserted-by":"crossref","first-page":"3286","DOI":"10.1093\/brain\/awab398","article-title":"Hitching a ride on exosomes: a new approach for the delivery of siRNA-mediated therapies","volume":"144","author":"Snyder","year":"2021","journal-title":"Brain"},{"key":"10.1016\/j.omtn.2024.102442_bib87","doi-asserted-by":"crossref","first-page":"467","DOI":"10.1261\/rna.054163.115","article-title":"A dual fluorescent reporter for the investigation of methionine mistranslation in live cells","volume":"22","author":"Gomes","year":"2016","journal-title":"RNA"},{"key":"10.1016\/j.omtn.2024.102442_bib88","article-title":"Screening for amyloid aggregation by Semi-Denaturing Detergent-Agarose Gel Electrophoresis","volume":"17","author":"Halfmann","year":"2008","journal-title":"J. Vis. Exp."}],"container-title":["Molecular Therapy Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253124003299?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253124003299?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,8]],"date-time":"2025-11-08T11:28:24Z","timestamp":1762601304000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253124003299"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,3]]},"references-count":91,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2025,3]]}},"alternative-id":["S2162253124003299"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2024.102442","relation":{},"ISSN":["2162-2531"],"issn-type":[{"value":"2162-2531","type":"print"}],"subject":[],"published":{"date-parts":[[2025,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Glutamine missense suppressor transfer RNAs inhibit polyglutamine aggregation","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2024.102442","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"102442"}}