{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,25]],"date-time":"2026-09-25T02:21:05Z","timestamp":1790302865009,"version":"4.1.0"},"reference-count":370,"publisher":"The Company of Biologists","issue":"10","license":[{"start":{"date-parts":[[2024,11,6]],"date-time":"2024-11-06T00:00:00Z","timestamp":1730851200000},"content-version":"vor","delay-in-days":36,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100000065","name":"National Institute of Neurological Disorders and Stroke","doi-asserted-by":"crossref","award":["R01NS134506"],"award-info":[{"award-number":["R01NS134506"]}],"id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100000069","name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases","doi-asserted-by":"crossref","award":["K08AR075894"],"award-info":[{"award-number":["K08AR075894"]}],"id":[{"id":"10.13039\/100000069","id-type":"DOI","asserted-by":"crossref"}]},{"name":"American Society of Gene and Cell Therapy"},{"DOI":"10.13039\/100018235","name":"St. Louis Children's Hospital","doi-asserted-by":"crossref","award":["MIFR20221004"],"award-info":[{"award-number":["MIFR20221004"]}],"id":[{"id":"10.13039\/100018235","id-type":"DOI","asserted-by":"crossref"}]},{"name":"LGMD 1D DNAJB6 Foundation and International Registry"},{"name":"Cure HSPB8"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2024,10,1]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n               <jats:p>Treatments for disabling and life-threatening hereditary muscle disorders are finally close to becoming a reality. Research has thus far focused primarily on recessive forms of muscle disease. The gene replacement strategies that are commonly employed for recessive, loss-of-function disorders are not readily translatable to most dominant myopathies owing to the presence of a normal chromosome in each nucleus, hindering the development of novel treatments for these dominant disorders. This is largely due to their complex, heterogeneous disease mechanisms that require unique therapeutic approaches. However, as viral and RNA interference-based therapies enter clinical use, key tools are now in place to develop treatments for dominantly inherited disorders of muscle. This article will review what is known about dominantly inherited disorders of muscle, specifically their genetic basis, how mutations lead to disease, and the pathomechanistic implications for therapeutic approaches.<\/jats:p>","DOI":"10.1242\/dmm.050720","type":"journal-article","created":{"date-parts":[[2024,11,6]],"date-time":"2024-11-06T07:29:57Z","timestamp":1730878197000},"source":"Crossref","is-referenced-by-count":9,"title":["Dominantly inherited muscle disorders: understanding their complexity and exploring therapeutic approaches"],"prefix":"10.1242","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8728-9833","authenticated-orcid":false,"given":"Andrew R.","family":"Findlay","sequence":"first","affiliation":[{"name":"Washington University Saint Louis , Neuromuscular Disease Center, 660 S. 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