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Autosomal dominant mutations V133F and Y330C in histidyl-tRNA synthetase (HARS) cause the genetic disorder Charcot-Marie-Tooth type 2W (CMT2W). Treatments are currently restricted to symptom relief, with no therapeutic available that targets the cause of disease. We previously found that histidine supplementation alleviated phenotypic defects in a humanized yeast model of CMT2W caused by HARS V155G and S356N that also unexpectedly exacerbated the phenotype of the two HARS mutants V133F and Y330C. Here, we show that V133F destabilizes recombinant HARS protein, which is rescued in the presence of tRNAHis. HARS V133F and Y330C cause mistranslation and cause changes to the proteome without activating the integrated stress response as validated by mass spectrometry and growth defects that persist with histidine supplementation. The growth defects and reduced translation fidelity caused by V133F and Y330C mutants were rescued by supplementation with human tRNAHis in a humanized yeast model. Our results demonstrate the feasibility of cognate tRNA as a therapeutic that rescues HARS deficiency and ameliorates toxic mistranslation generated by causative alleles for CMT.<\/jats:p>","DOI":"10.1093\/nar\/gkae996","type":"journal-article","created":{"date-parts":[[2024,10,16]],"date-time":"2024-10-16T15:45:14Z","timestamp":1729093514000},"page":"14043-14060","source":"Crossref","is-referenced-by-count":10,"title":["Transfer RNA supplementation rescues HARS deficiency in a humanized yeast model of Charcot-Marie-Tooth disease"],"prefix":"10.1093","volume":"52","author":[{"given":"Sarah\u00a0D P","family":"Wilhelm","sequence":"first","affiliation":[{"name":"Department of Biochemistry, The University of Western Ontario , London , Ontario N6A 5C1 ,","place":["Canada"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jenica\u00a0H","family":"Kakadia","sequence":"additional","affiliation":[{"name":"Department of Biochemistry, The University of Western Ontario , London , Ontario N6A 5C1 ,","place":["Canada"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aruun","family":"Beharry","sequence":"additional","affiliation":[{"name":"Department of Biochemistry, The University of Western Ontario , London , Ontario N6A 5C1 ,","place":["Canada"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rosan","family":"Kenana","sequence":"additional","affiliation":[{"name":"Department of Biochemistry, The University of Western Ontario , London , Ontario N6A 5C1 ,","place":["Canada"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kyle\u00a0S","family":"Hoffman","sequence":"additional","affiliation":[{"name":"Bioinformatics Solutions Inc , Waterloo , Ontario ,\u00a0N2L 3K8","place":["Canada"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9500-8640","authenticated-orcid":false,"given":"Patrick","family":"O\u2019Donoghue","sequence":"additional","affiliation":[{"name":"Department of Biochemistry, The University of Western Ontario , London , Ontario N6A 5C1 ,","place":["Canada"]},{"name":"Department of Chemistry, The University of Western Ontario , London , Ontario N6A 5C1 ,","place":["Canada"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1721-1667","authenticated-orcid":false,"given":"Ilka\u00a0U","family":"Heinemann","sequence":"additional","affiliation":[{"name":"Department of Biochemistry, The University of Western Ontario , London , Ontario N6A 5C1 ,","place":["Canada"]},{"name":"Children's Health Research Institute , London , ON , N6C 4V3","place":["Canada"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2024,11,12]]},"reference":[{"key":"2024122115520369000_B1","doi-asserted-by":"crossref","first-page":"3419","DOI":"10.3390\/ijms20143419","article-title":"Charcot-Marie-Tooth: from molecules to therapy","volume":"20","author":"Morena","year":"2019","journal-title":"Int. J. Mol. Sci."},{"key":"2024122115520369000_B2","doi-asserted-by":"crossref","first-page":"e029240","DOI":"10.1136\/bmjopen-2019-029240","article-title":"Prevalence of Charcot-Marie-Tooth disease across the lifespan: a population-based epidemiological study","volume":"9","author":"Theadom","year":"2019","journal-title":"BMJ Open"},{"key":"2024122115520369000_B3","doi-asserted-by":"crossref","first-page":"1293","DOI":"10.1086\/375039","article-title":"Glycyl tRNA synthetase mutations in Charcot-Marie-Tooth disease type 2D and distal spinal muscular atrophy type V","volume":"72","author":"Antonellis","year":"2003","journal-title":"Am. J. Hum. Genet."},{"key":"2024122115520369000_B4","doi-asserted-by":"crossref","first-page":"197","DOI":"10.1038\/ng1727","article-title":"Disrupted function and axonal distribution of mutant tyrosyl-tRNA synthetase in dominant intermediate Charcot-Marie-Tooth neuropathy","volume":"38","author":"Jordanova","year":"2006","journal-title":"Nat. Genet."},{"key":"2024122115520369000_B5","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1016\/j.ajhg.2009.12.005","article-title":"A major determinant for binding and aminoacylation of tRNA (Ala) in cytoplasmic Alanyl-tRNA synthetase is mutated in dominant axonal Charcot-Marie-Tooth disease","volume":"86","author":"Latour","year":"2010","journal-title":"Am. J. Hum. Genet."},{"key":"2024122115520369000_B6","doi-asserted-by":"crossref","first-page":"2161","DOI":"10.1093\/brain\/awv158","article-title":"Loss of function mutations in HARS cause a spectrum of inherited peripheral neuropathies","volume":"138","author":"Safka\u00a0Brozkova","year":"2015","journal-title":"Brain"},{"key":"2024122115520369000_B7","doi-asserted-by":"crossref","first-page":"1247","DOI":"10.1136\/jnnp-2013-305049","article-title":"Exome sequencing identifies a significant variant in methionyl-tRNA synthetase (MARS) in a family with late-onset CMT2","volume":"84","author":"Gonzalez","year":"2013","journal-title":"J. Neurol. Neurosurg. 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Application to studying recognition of Escherichia coli initiator tRNA mutants by glutaminyl-tRNA synthetase","volume":"266","author":"Varshney","year":"1991","journal-title":"J. Biol. Chem."},{"key":"2024122115520369000_B30","doi-asserted-by":"crossref","first-page":"2076","DOI":"10.1091\/mbc.e13-01-0010","article-title":"Ubiquitin conjugation triggers misfolded protein sequestration into quality control foci when Hsp70 chaperone levels are limiting","volume":"24","author":"Shiber","year":"2013","journal-title":"Mol. Biol. 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