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Although multiple translational readthrough-inducing drugs (TRIDs) have been reported, their relative efficacy remains inconsistent across studies, likely due to differences in experimental systems and cellular contexts. A systematic comparison across complementary and clinically relevant models is therefore required to better define their therapeutic potential. This study aims to evaluate and compare the efficiency of different TRIDs used for nonsense mutation correction.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We performed a comparative evaluation of 12 TRIDs using a panel of cellular models encompassing increasing levels of physiological relevance. These included a firefly luciferase-based reporter assay, human cell lines harboring endogenous TP53 nonsense mutations, patient-derived intestinal organoids carrying CFTR nonsense variants, and Fischer rat thyroid (FRT) cells expressing homozygous CFTR nonsense mutations. Readthrough efficiency was assessed and compared across models.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Across the different systems, 2,6-diaminopurine (DAP), clitocine, SRI-41315, and TLN468 consistently exhibited the highest readthrough activity. However, variations in relative efficacy were observed depending on the assay and cellular context, underscoring the influence of experimental conditions, amino acid incorporation profiles, and cell-specific determinants on readthrough outcomes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Our findings demonstrate the necessity of evaluating candidate TRIDs across multiple complementary and patient-relevant models to accurately estimate their therapeutic potential. This study identifies several promising compounds for further preclinical development and provides a comparative framework to guide the rational selection of readthrough-inducing agents for translational applications.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1186\/s12967-026-08058-5","type":"journal-article","created":{"date-parts":[[2026,3,31]],"date-time":"2026-03-31T11:55:36Z","timestamp":1774958136000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Comparative evaluation of TRIDs : a strategy to improve treatments"],"prefix":"10.1186","volume":"24","author":[{"given":"Fatima","family":"Hariss","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sacha","family":"Spelier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Louise","family":"Damy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmen Sandoval","family":"Pacheco","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luca","family":"Houpe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Leroy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xavier","family":"Thuru","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fr\u00e9d\u00e9ric","family":"Becq","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5132-3585","authenticated-orcid":false,"given":"Fabrice","family":"Lejeune","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,3,31]]},"reference":[{"key":"8058_CR1","doi-asserted-by":"publisher","first-page":"333","DOI":"10.1261\/rna.080334.124","volume":"31","author":"M M","year":"2025","unstructured":"McMahon M and Maquat LE. 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The study was conducted in accordance with the Declaration of Helsinki, and approved by a specific ethical board for the use of biobanked materials TcBIO (Toetsingscommissie Biobanks, TcBio#19\u2013831, release protocol 24\u2013263), an institutional Medical Research Ethics Committee of the University Medical Center Utrecht. Informed consent for intestinal tissue collection, generation, storage and use of the organoids was obtained from all participating patients. Biobanked intestinal organoids are stored and catalogued (\n                      \n                      ) at the foundation Hubrecht Organoid Technology (\n                      \n                      ) and can be requested at info@huborganoids.nl.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"All authors of this study have given their permission to publish their results.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"FL is a cofounder of Genvade Therapeutics that develops 2,6-diaminopurine.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interest"}}],"article-number":"661"}}