{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,24]],"date-time":"2026-10-24T08:03:21Z","timestamp":1792829001779,"version":"4.5.0"},"reference-count":47,"publisher":"American Association for the Advancement of Science (AAAS)","issue":"37","funder":[{"name":"Tevard Biosciences, Inc."}],"content-domain":{"domain":["www.science.org"],"crossmark-restriction":true},"short-container-title":["Sci. Adv."],"published-print":{"date-parts":[[2026,9,11]]},"abstract":"<jats:p>Duchenne muscular dystrophy (DMD) is a fatal disorder caused by loss of dystrophin, a protein essential for muscle cell integrity. To date, no therapeutic has restored full-length dystrophin. Suppressor transfer RNAs (sup-tRNAs) have the potential to treat the \u223c15% of patients with DMD and nonsense mutations. We have evolved highly potent UAA sup-tRNAs through an extensive mutagenesis screening campaign, optimized our adeno-associated virus (AAV) expression vectors, and developed efficient RNA polymerase III promoters. We show that systemic delivery of these sup-tRNAs restores full-length dystrophin and recovers muscle strength and motor function in mice at lower doses than AAV microdystrophin therapies. We see no adverse toxicology and observe proteome-wide reversion of molecular pathophysiology. These data establish sup-tRNAs as a promising therapeutic platform for DMD and other disorders.<\/jats:p>","DOI":"10.1126\/sciadv.aeg3466","type":"journal-article","created":{"date-parts":[[2026,9,9]],"date-time":"2026-09-09T17:58:47Z","timestamp":1788976727000},"update-policy":"https:\/\/doi.org\/10.34133\/aaas_crossmark","source":"Crossref","is-referenced-by-count":1,"title":["Engineering suppressor tRNAs for effective treatment of Duchenne muscular dystrophy"],"prefix":"10.1126","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6648-3883","authenticated-orcid":true,"given":"Julien","family":"Oury","sequence":"first","affiliation":[{"name":"Tevard Biosciences, Lilly Gateway Labs, 15 Necco St., Boston, MA, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5868-0238","authenticated-orcid":true,"given":"Ying-Hsin","family":"Chen","sequence":"additional","affiliation":[{"name":"Tevard Biosciences, Lilly Gateway Labs, 15 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