{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Porter JJ"],"funding":["Cystic Fibrosis Foundation","NIH HHS"],"pagination":["14112-14132"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11662937"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["52(22)"],"pubmed_abstract":["Nonsense suppressor transfer RNAs (tRNAs) or AntiCodon-Edited tRNAs (ACE-tRNAs) have long been envisioned as a therapeutic approach to overcome genetic diseases resulting from the introduction of premature termination codons (PTCs). The ACE-tRNA approach for the rescue of PTCs has been hampered by ineffective delivery through available modalities for gene therapy. Here we have screened a series of ACE-tRNA expression cassette sequence libraries containing >1800 members in an effort to optimize ACE-tRNA function and provide a roadmap for optimization in the future. By optimizing PTC suppression efficiency of ACE-tRNAs, we have decreased the amount of ACE-tRNA required by ∼16-fold for the most common cystic fibrosis-causing PTCs."],"journal":["Nucleic acids research"],"pubmed_title":["Optimization of ACE-tRNAs function in translation for suppression of nonsense mutations."],"pmcid":["PMC11662937"],"funding_grant_id":["PORTER20F0","R01 HL153988"],"pubmed_authors":["Porter JJ","Lueck JD","Ko W","Sorensen EG"],"additional_accession":[]},"is_claimable":false,"name":"Optimization of ACE-tRNAs function in translation for suppression of nonsense mutations.","description":"Nonsense suppressor transfer RNAs (tRNAs) or AntiCodon-Edited tRNAs (ACE-tRNAs) have long been envisioned as a therapeutic approach to overcome genetic diseases resulting from the introduction of premature termination codons (PTCs). The ACE-tRNA approach for the rescue of PTCs has been hampered by ineffective delivery through available modalities for gene therapy. Here we have screened a series of ACE-tRNA expression cassette sequence libraries containing >1800 members in an effort to optimize ACE-tRNA function and provide a roadmap for optimization in the future. By optimizing PTC suppression efficiency of ACE-tRNAs, we have decreased the amount of ACE-tRNA required by ∼16-fold for the most common cystic fibrosis-causing PTCs.","dates":{"release":"2024-01-01T00:00:00Z","publication":"2024 Dec","modification":"2025-04-04T21:11:33.915Z","creation":"2025-04-04T21:11:33.915Z"},"accession":"S-EPMC11662937","cross_references":{"pubmed":["39673265"],"doi":["10.1093/nar/gkae1112"]}}