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PTCs result in little to no full-length protein production and decreased mRNA stability due to the nonsense-mediated mRNA decay (NMD) pathway. We provide evidence that anticodon-edited (ACE-) tRNAs efficiently suppress the most prevalent cystic fibrosis (CF)-causing PTCs, promoting significant rescue of endogenous cystic fibrosis transmembrane conductance regulator (CFTR) transcript abundance and channel function in different model systems. We show that our best-performing ACE-tRNA, which decodes all UGA PTCs to a leucine amino acid, markedly rescues CFTR function from the most prevalent CF-causing PTCs, all of which arose from nonleucine encoding codons. Using this single ACE-tRNA variant, we demonstrate significant rescue of CFTR function in an immortalized airway cell line and two different primary CF patient-derived intestinal cell models with CFTR nonsense mutations. Further, we demonstrate that leucine substitution CFTR variants are highly functional. Thus, ACE-tRNAs have promise as a platform therapeutic for CF and other nonsense-associated diseases.<\/jats:p>","DOI":"10.1093\/nar\/gkaf675","type":"journal-article","created":{"date-parts":[[2025,7,12]],"date-time":"2025-07-12T16:40:53Z","timestamp":1752338453000},"source":"Crossref","is-referenced-by-count":14,"title":["ACE-tRNAs are a platform technology for suppressing nonsense mutations that cause cystic fibrosis"],"prefix":"10.1093","volume":"53","author":[{"given":"Wooree","family":"Ko","sequence":"first","affiliation":[{"name":"University of Rochester School of Medicine and Dentistry Department of Pharmacology and Physiology, , Rochester , NY\u00a0 14642 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4542-0338","authenticated-orcid":false,"given":"Joseph\u00a0J","family":"Porter","sequence":"additional","affiliation":[{"name":"University of Rochester School of Medicine and Dentistry Department of 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Utrecht University , 3584\u00a0 CT , Utrecht ,","place":["The Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tyler","family":"Couch","sequence":"additional","affiliation":[{"name":"University of Rochester School of Medicine and Dentistry Department of Pharmacology and Physiology, , Rochester , NY\u00a0 14642 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kevin","family":"Coote","sequence":"additional","affiliation":[{"name":"Cystic Fibrosis Foundation Therapeutics Lab, Cystic Fibrosis Foundation , Lexington , MA\u00a002421,\u00a0","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin","family":"Mense","sequence":"additional","affiliation":[{"name":"Cystic Fibrosis Foundation Therapeutics Lab, Cystic Fibrosis Foundation , Lexington , MA\u00a002421,\u00a0","place":["United 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