{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,19]],"date-time":"2026-08-19T20:15:28Z","timestamp":1787170528319,"version":"build-2736575974"},"reference-count":34,"publisher":"MDPI AG","issue":"6","license":[{"start":{"date-parts":[[2025,3,13]],"date-time":"2025-03-13T00:00:00Z","timestamp":1741824000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Association Fran\u00e7aise contre les myopathies","award":["24114"],"award-info":[{"award-number":["24114"]}]},{"name":"Institut National de la sant\u00e9 et la recherche m\u00e9dicale (INSERM)","award":["24114"],"award-info":[{"award-number":["24114"]}]},{"name":"Association Monegasque contre les myopathies (AMM)","award":["24114"],"award-info":[{"award-number":["24114"]}]},{"name":"Paris Ile-de-France Region and the Fondation UVSQ","award":["24114"],"award-info":[{"award-number":["24114"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by the progressive degeneration of skeletal and cardiac muscles due to the absence of dystrophin. Exon-skipping therapy is among the most promising approaches for treating DMD, with several antisense oligonucleotides (ASO) already approved by the FDA; however, their limited efficacy highlights substantial potential for further improvement. In this study, we evaluate the potential of combining ASO with valproic acid (VPA) to enhance dystrophin expression and improve functional outcomes in a murine model of DMD. Our results indicate that the ASO+VPA treatment significantly increases dystrophin restoration across various muscle tissues, with particularly pronounced effects observed in cardiac muscle, where levels are nearly doubled compared to ASO monotherapy. Additionally, we demonstrate significant improvements in functional outcomes in treated mdx mice. Our findings suggest that the combined ASO+VPA therapy holds promise as an effective therapeutic approach to ameliorate muscle function in DMD, warranting further exploration of its mechanistic pathways and long-term benefits.<\/jats:p>","DOI":"10.3390\/ijms26062583","type":"journal-article","created":{"date-parts":[[2025,3,13]],"date-time":"2025-03-13T06:53:00Z","timestamp":1741848780000},"page":"2583","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Valproic Acid Improves Antisense-Mediated Exon-Skipping Efficacy in mdx Mice"],"prefix":"10.3390","volume":"26","author":[{"given":"Micky","family":"Phongsavanh","sequence":"first","affiliation":[{"name":"Universit\u00e9 Paris-Saclay, UVSQ, Inserm, END-ICAP, 78000 Versailles, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1968-3596","authenticated-orcid":false,"given":"Flavien","family":"Bizot","sequence":"additional","affiliation":[{"name":"Universit\u00e9 Paris-Saclay, UVSQ, Inserm, END-ICAP, 78000 Versailles, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amel","family":"Saoudi","sequence":"additional","affiliation":[{"name":"Universit\u00e9 Paris-Saclay, UVSQ, Inserm, END-ICAP, 78000 Versailles, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-4813-1080","authenticated-orcid":false,"given":"Cecile","family":"Gastaldi","sequence":"additional","affiliation":[{"name":"Medical Biology Department, Centre Scientifique de Monaco, 98000 Monaco, Monaco"},{"name":"LIA BAHN, CSM-UVSQ, 98000 Monaco, Monaco"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olivier","family":"Le Coz","sequence":"additional","affiliation":[{"name":"Universit\u00e9 Paris-Saclay, UVSQ, Inserm, END-ICAP, 78000 Versailles, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Tensorer","sequence":"additional","affiliation":[{"name":"SQY Therapeutics, UVSQ, 78180 Montigny le Bretonneux, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8740-5136","authenticated-orcid":false,"given":"Elise","family":"Brisebard","sequence":"additional","affiliation":[{"name":"INRAE Oniris, UMR 703 PAnTher, 44300 Nantes, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luis","family":"Garcia","sequence":"additional","affiliation":[{"name":"Universit\u00e9 Paris-Saclay, UVSQ, Inserm, END-ICAP, 78000 Versailles, France"},{"name":"Medical Biology Department, Centre Scientifique de Monaco, 98000 Monaco, Monaco"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3938-1165","authenticated-orcid":false,"given":"Aur\u00e9lie","family":"Goyenvalle","sequence":"additional","affiliation":[{"name":"Universit\u00e9 Paris-Saclay, UVSQ, Inserm, END-ICAP, 78000 Versailles, France"},{"name":"Medical Biology Department, Centre Scientifique de Monaco, 98000 Monaco, Monaco"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,3,13]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1038\/s41572-021-00248-3","article-title":"Duchenne Muscular Dystrophy","volume":"7","author":"Duan","year":"2021","journal-title":"Nat. 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