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Nonsense suppressor anticodon-edited transfer RNAs (ACE-tRNAs) have emerged as a therapeutic modality for the rescue of PTCs. Delivery of ACE-tRNAs\u00a0in vivo has been achieved by adeno-associated viral vector and RNA-lipid nanoparticle; however, due to drawbacks associated with these approaches, DNA delivery remains an attractive approach. DNA-based approaches afford ease of manufacturing at a relatively low cost and exhibit improved therapeutic durability and safety as compared to viral vector- or RNA-based approaches. Due to the small size of human tRNA genes employed as ACE-tRNAs, in principle, DNA vectors &amp;lt;200 base pairs (bp) in size (minivectors) could be utilized for delivery of actively transcribed ACE-tRNAs. Here, we demonstrate that linear DNA ACE-tRNA vectors as small as 200 bp effectively suppress several nonsense mutations in CFTR and REP1, and that ACE-tRNA minivectors, when tested in cell or ex vivo models, display significantly improved bioavailability, reduced innate immune burden, and superior biostability as compared to conventional plasmid DNA vectors.<\/jats:p>","DOI":"10.1093\/nar\/gkag082","type":"journal-article","created":{"date-parts":[[2026,1,23]],"date-time":"2026-01-23T12:38:24Z","timestamp":1769171904000},"source":"Crossref","is-referenced-by-count":4,"title":["Anticodon-edited transfer RNAs (ACE-tRNAs) encoded as therapeutic nonviral minimal DNA vectors"],"prefix":"10.1093","volume":"54","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4542-0338","authenticated-orcid":false,"given":"Joseph J","family":"Porter","sequence":"first","affiliation":[{"name":"Department of Pharmacology and Physiology, University of Rochester School of Medicine and Dentistry , Rochester , 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K","family":"Peterson","sequence":"additional","affiliation":[{"name":"Department of Pharmacology and Physiology, University of Rochester School of Medicine and Dentistry , Rochester , NY 14642 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7748-8073","authenticated-orcid":false,"given":"David A","family":"Dean","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Rochester School of Medicine and Dentistry , Rochester , NY 14642 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1820-711X","authenticated-orcid":false,"given":"John D","family":"Lueck","sequence":"additional","affiliation":[{"name":"Department of Pharmacology and Physiology, University of Rochester School of Medicine and Dentistry , Rochester , NY 14642 ,","place":["United States"]},{"name":"Department of Neurology, University of Rochester 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