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A facile and economical oral liquid formulation is developed by converting UAAs into ionic liquids, significantly enhancing their bioavailability and tissue accumulation. Empirical data reveal a 10\u2010fold increase in bioavailability and up to a 13\u2010fold rise in focal tissue accumulation, alongside marked improvements in UAA incorporation efficiency. A 4\u2010week oral administration in\n                    <jats:italic>mdx<\/jats:italic>\n                    mice, a model for Duchenne muscular dystrophy (DMD), demonstrates the formulation's unprecedented therapeutic potential, with up to 40% dystrophin expression restoration and 75% recovery of normal fiber functions, surpassing existing treatments and exhibiting substantial long\u2010term safety. This study presents a potent oral dosage form that dramatically improves UAA incorporation into target proteins in vivo, offering a significant advance in the treatment of nonsense mutation\u2010mediated disorders and holding considerable promise for clinical translation.\n                  <\/jats:p>","DOI":"10.1002\/advs.202306792","type":"journal-article","created":{"date-parts":[[2024,1,30]],"date-time":"2024-01-30T03:54:00Z","timestamp":1706586840000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Unnatural Amino Acid\u2010Based Ionic Liquid Enables Oral Treatment of Nonsense Mutation Disease in Mice"],"prefix":"10.1002","volume":"11","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7610-0433","authenticated-orcid":false,"given":"Yujie","family":"Shi","sequence":"first","affiliation":[{"name":"State Key Laboratory of Natural and Biomimetic Drugs Peking University  Beijing 100191 China"},{"name":"Department 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