{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T08:07:39Z","timestamp":1790842059499,"version":"4.1.0"},"reference-count":71,"publisher":"National Academy of Sciences","issue":"2","license":[{"start":{"date-parts":[[2023,1,3]],"date-time":"2023-01-03T00:00:00Z","timestamp":1672704000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"name":"Association Monegasque contre les Myopathies","award":["00"],"award-info":[{"award-number":["00"]}]},{"name":"Association Francaise contre les Myopathies","award":["23802"],"award-info":[{"award-number":["23802"]}]},{"name":"Universite Franco-Allemande","award":["CDFA-06-11"],"award-info":[{"award-number":["CDFA-06-11"]}]},{"DOI":"10.13039\/501100001665","name":"Agence Nationale de la Recherche","doi-asserted-by":"publisher","award":["ANR-16-CE14-0002"],"award-info":[{"award-number":["ANR-16-CE14-0002"]}],"id":[{"id":"10.13039\/501100001665","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["www.pnas.org"],"crossmark-restriction":true},"short-container-title":["Proc. Natl. Acad. Sci. U.S.A."],"published-print":{"date-parts":[[2023,1,10]]},"abstract":"<jats:p>\n                    Dystrophin is essential for muscle health: its sarcolemmal absence causes the fatal, X-linked condition, Duchenne muscular dystrophy (DMD). However, its normal, spatial organization remains poorly understood, which hinders the interpretation of efficacy of its therapeutic restoration. Using female reporter mice heterozygous for fluorescently tagged dystrophin (\n                    <jats:italic>\n                      Dmd\n                      <jats:sup>EGFP<\/jats:sup>\n                    <\/jats:italic>\n                    ), we here reveal that dystrophin distribution is unexpectedly compartmentalized, being restricted to myonuclear-defined sarcolemmal territories extending ~80 \u00b5m, which we called \u201cbasal sarcolemmal dystrophin units (BSDUs).\u201d These territories were further specialized at myotendinous junctions, where both\n                    <jats:italic>Dmd<\/jats:italic>\n                    transcripts and dystrophin protein were enriched. Genome-level correction in X-linked muscular dystrophy mice via CRISPR\/Cas9 gene editing restored a mosaic of separated dystrophin domains, whereas transcript-level\n                    <jats:italic>Dmd<\/jats:italic>\n                    correction, following treatment with tricyclo-DNA\u00a0antisense oligonucleotides, restored dystrophin initially at junctions before extending along the entire fiber\u2014with levels ~2% sufficient to moderate the dystrophic process. We conclude that widespread restoration of fiber dystrophin is likely critical for therapeutic success in DMD, perhaps most importantly, at muscle\u2013tendon junctions.\n                  <\/jats:p>","DOI":"10.1073\/pnas.2206324120","type":"journal-article","created":{"date-parts":[[2023,1,3]],"date-time":"2023-01-03T15:21:36Z","timestamp":1672759296000},"update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":39,"title":["Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle"],"prefix":"10.1073","volume":"120","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4231-3112","authenticated-orcid":false,"given":"Adrien","family":"Morin","sequence":"first","affiliation":[{"name":"Evolution of Neuromuscular Diseases - Innovative Concepts and Practice (END-ICAP) U1179, Universit\u00e9 Paris-Saclay, Universit\u00e9 de Versailles Saint-Quentin-en-Yvelines, Inserm, 78000 Versailles, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4903-6384","authenticated-orcid":false,"given":"Amalia","family":"Stantzou","sequence":"additional","affiliation":[{"name":"Evolution of Neuromuscular Diseases - Innovative Concepts and Practice (END-ICAP) U1179, Universit\u00e9 Paris-Saclay, Universit\u00e9 de Versailles Saint-Quentin-en-Yvelines, Inserm, 78000 Versailles, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olga N.","family":"Petrova","sequence":"additional","affiliation":[{"name":"Evolution of Neuromuscular Diseases - Innovative Concepts and Practice (END-ICAP) U1179, Universit\u00e9 Paris-Saclay, Universit\u00e9 de Versailles Saint-Quentin-en-Yvelines, Inserm, 78000 Versailles, 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