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The absence of protein expression and the consequent loss of essential cellular functions are responsible for the severe phenotypes in the so\u2010called genetic nonsense\u2010related diseases (NRDs), such as cystic fibrosis, hemophilia, Duchenne muscular dystrophy, Fabry disease, Choroideremia, Usher syndrome, Shwachman\u2013Diamond syndrome, and even certain types of cancer. Nonsense mutations pose a significant challenge in the treatment of NRDs, as a specific approach directly addressing this genetic defect is currently unavailable. Developing new therapeutic strategies for nonsense suppression is a crucial goal of precision medicine. This review describes some of the most promising therapeutic approaches and emerging strategies for treating NRDs. 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Future research should focus on refining these approaches and exploring integrated and personalized treatments to enhance therapeutic outcomes and ensure continuous improvement in the quality of care.<\/jats:p>","DOI":"10.1002\/iub.70027","type":"journal-article","created":{"date-parts":[[2025,5,27]],"date-time":"2025-05-27T08:10:28Z","timestamp":1748333428000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Advancing Therapeutic Strategies for Nonsense\u2010Related Diseases: From Small Molecules to Nucleic Acid\u2010Based Innovations"],"prefix":"10.1002","volume":"77","author":[{"given":"Davide","family":"Ricci","sequence":"first","affiliation":[{"name":"Department of Biological, Chemical and Pharmaceutical Sciences and Technologies University of Palermo  Palermo 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