{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T23:33:57Z","timestamp":1793057637273,"version":"4.5.0"},"reference-count":27,"publisher":"National Academy of Sciences","issue":"43","license":[{"start":{"date-parts":[[2019,4,5]],"date-time":"2019-04-05T00:00:00Z","timestamp":1554422400000},"content-version":"vor","delay-in-days":182,"URL":"http:\/\/www.pnas.org\/site\/aboutpnas\/licenses.xhtml"}],"content-domain":{"domain":["www.pnas.org"],"crossmark-restriction":true},"short-container-title":["Proc. Natl. Acad. Sci. U.S.A."],"published-print":{"date-parts":[[2018,10,23]]},"abstract":"<jats:title>Significance<\/jats:title>\n                  <jats:p>\n                    Centronuclear myopathies are rare and severe congenital muscle diseases. Here we hypothesized that reducing dynamin 2 (DNM2) may rescue the pathophysiology observed in\n                    <jats:italic>DNM2<\/jats:italic>\n                    -related dominant centronuclear myopathy. The total DNM2 expression was reduced in a faithful murine model (\n                    <jats:italic>Dnm2<\/jats:italic>\n                    <jats:sup>RW\/+<\/jats:sup>\n                    mice) using two different methods targeting both mutated and wild-type\n                    <jats:italic>Dnm2<\/jats:italic>\n                    , adeno-associated virus-shRNA, or antisense oligonucleotides, leading to a restoration of muscle mass, histopathology, and muscle ultrastructural features to wild-type levels. This provides a therapeutic strategy for treating this disease. We also propose that targeting both alleles in dominant diseases due to a mutation in only one of the alleles can successfully rescue the phenotypes.\n                  <\/jats:p>","DOI":"10.1073\/pnas.1808170115","type":"journal-article","created":{"date-parts":[[2018,10,8]],"date-time":"2018-10-08T09:58:50Z","timestamp":1538992730000},"page":"11066-11071","update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":76,"title":["Reducing dynamin 2 (DNM2) rescues\n                    <i>DNM2<\/i>\n                    -related dominant centronuclear myopathy"],"prefix":"10.1073","volume":"115","author":[{"given":"Suzie","family":"Buono","sequence":"first","affiliation":[{"name":"Institut de G\u00e9n\u00e9tique et de Biologie Mol\u00e9culaire et Cellulaire, 67404 Illkirch, France;"},{"name":"Institut National de la Sant\u00e9 et de la Recherche M\u00e9dicale, U1258, 67404 Illkirch, France;"},{"name":"Centre National de la Recherche Scientifique, UMR7104, Illkirch, 67404, France;"},{"name":"Strasbourg University, 67404 Illkirch, France;"},{"name":"Dynacure, 67400 Illkirch, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacob A.","family":"Ross","sequence":"additional","affiliation":[{"name":"Centre of Human and Applied Physiological Sciences, School of Basic and Medical Biosciences, Faculty of Life Sciences and Medicine, King\u2019s College London, SE1 1UL London, United Kingdom;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hichem","family":"Tasfaout","sequence":"additional","affiliation":[{"name":"Institut de G\u00e9n\u00e9tique et de Biologie Mol\u00e9culaire et Cellulaire, 67404 Illkirch, France;"},{"name":"Institut National de la Sant\u00e9 et de la Recherche M\u00e9dicale, U1258, 67404 Illkirch, France;"},{"name":"Centre National de la Recherche Scientifique, UMR7104, Illkirch, 67404, France;"},{"name":"Strasbourg University, 67404 Illkirch, 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