{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T15:52:28Z","timestamp":1789055548838,"version":"build-2803163510"},"reference-count":38,"publisher":"MDPI AG","issue":"20","license":[{"start":{"date-parts":[[2023,10,11]],"date-time":"2023-10-11T00:00:00Z","timestamp":1696982400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"European Union-NextGenerationEU","award":["PNRR-M4C2-I1.3"],"award-info":[{"award-number":["PNRR-M4C2-I1.3"]}]},{"name":"European Union-NextGenerationEU","award":["B73C22001250006"],"award-info":[{"award-number":["B73C22001250006"]}]},{"name":"European Union-NextGenerationEU","award":["FFC#06\/2020"],"award-info":[{"award-number":["FFC#06\/2020"]}]},{"name":"Project PE_00000019 \u201cHEAL ITALIA\u201d","award":["PNRR-M4C2-I1.3"],"award-info":[{"award-number":["PNRR-M4C2-I1.3"]}]},{"name":"Project PE_00000019 \u201cHEAL ITALIA\u201d","award":["B73C22001250006"],"award-info":[{"award-number":["B73C22001250006"]}]},{"name":"Project PE_00000019 \u201cHEAL ITALIA\u201d","award":["FFC#06\/2020"],"award-info":[{"award-number":["FFC#06\/2020"]}]},{"DOI":"10.13039\/501100008385","name":"Italian Cystic Fibrosis Research Foundation","doi-asserted-by":"publisher","award":["PNRR-M4C2-I1.3"],"award-info":[{"award-number":["PNRR-M4C2-I1.3"]}],"id":[{"id":"10.13039\/501100008385","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100008385","name":"Italian Cystic Fibrosis Research Foundation","doi-asserted-by":"publisher","award":["B73C22001250006"],"award-info":[{"award-number":["B73C22001250006"]}],"id":[{"id":"10.13039\/501100008385","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100008385","name":"Italian Cystic Fibrosis Research Foundation","doi-asserted-by":"publisher","award":["FFC#06\/2020"],"award-info":[{"award-number":["FFC#06\/2020"]}],"id":[{"id":"10.13039\/501100008385","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Nonsense mutations cause several genetic diseases such as cystic fibrosis, Duchenne muscular dystrophy, \u03b2-thalassemia, and Shwachman\u2013Diamond syndrome. These mutations induce the formation of a premature termination codon (PTC) inside the mRNA sequence, resulting in the synthesis of truncated polypeptides. Nonsense suppression therapy mediated by translational readthrough-inducing drugs (TRIDs) is a promising approach to correct these genetic defects. TRIDs generate a ribosome miscoding of the PTC named \u201ctranslational readthrough\u201d and restore the synthesis of full-length and potentially functional proteins. The new oxadiazole-core TRIDs NV848, NV914, and NV930 (NV) showed translational readthrough activity in nonsense-related in vitro systems. In this work, the possible off-target effect of NV molecules on natural termination codons (NTCs) was investigated. Two different in vitro approaches were used to assess if the NV molecule treatment induces NTC readthrough: (1) a study of the translational-induced p53 molecular weight and functionality; (2) the evaluation of two housekeeping proteins\u2019 (Cys-C and \u03b22M) molecular weights. Our results showed that the treatment with NV848, NV914, or NV930 did not induce any translation alterations in both experimental systems. The data suggested that NV molecules have a specific action for the PTCs and an undetectable effect on the NTCs.<\/jats:p>","DOI":"10.3390\/ijms242015084","type":"journal-article","created":{"date-parts":[[2023,10,11]],"date-time":"2023-10-11T08:07:12Z","timestamp":1697011632000},"page":"15084","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Readthrough Approach Using NV Translational Readthrough-Inducing Drugs (TRIDs): A Study of the Possible Off-Target Effects on Natural Termination Codons (NTCs) on TP53 and Housekeeping Gene Expression"],"prefix":"10.3390","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1127-0391","authenticated-orcid":false,"given":"Riccardo","family":"Perriera","sequence":"first","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emanuele","family":"Vitale","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5885-164X","authenticated-orcid":false,"given":"Ivana","family":"Pibiri","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pietro Salvatore","family":"Carollo","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Davide","family":"Ricci","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federica","family":"Corrao","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ignazio","family":"Fiduccia","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4463-8156","authenticated-orcid":false,"given":"Raffaella","family":"Melfi","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Grazia","family":"Zizzo","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5059-8686","authenticated-orcid":false,"given":"Marco","family":"Tutone","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche 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