{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T08:07:05Z","timestamp":1790842025872,"version":"4.1.0"},"reference-count":61,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2023,5,23]],"date-time":"2023-05-23T00:00:00Z","timestamp":1684800000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100006769","name":"Russian Science Foundation","doi-asserted-by":"publisher","award":["21-75-00084"],"award-info":[{"award-number":["21-75-00084"]}],"id":[{"id":"10.13039\/501100006769","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Mutations that prevent the production of proteins in the DMD gene cause Duchenne muscular dystrophy. Most frequently, these are deletions leading to reading-frame shift. The \u201creading-frame rule\u201d states that deletions that preserve ORF result in a milder Becker muscular dystrophy. By removing several exons, new genome editing tools enable reading-frame restoration in DMD with the production of BMD-like dystrophins. However, not every truncated dystrophin with a significant internal loss functions properly. To determine the effectiveness of potential genome editing, each variant should be carefully studied in vitro or in vivo. In this study, we focused on the deletion of exons 8\u201350 as a potential reading-frame restoration option. Using the CRISPR-Cas9 tool, we created the novel mouse model DMDdel8-50, which has an in-frame deletion in the DMD gene. We compared DMDdel8-50 mice to C57Bl6\/CBA background control mice and previously generated DMDdel8-34 KO mice. We discovered that the shortened protein was expressed and correctly localized on the sarcolemma. The truncated protein, on the other hand, was unable to function like a full-length dystrophin and prevent disease progression. On the basis of protein expression, histological examination, and physical assessment of the mice, we concluded that the deletion of exons 8\u201350 is an exception to the reading-frame rule.<\/jats:p>","DOI":"10.3390\/ijms24119117","type":"journal-article","created":{"date-parts":[[2023,5,23]],"date-time":"2023-05-23T03:20:06Z","timestamp":1684812006000},"page":"9117","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["In-Frame Deletion of Dystrophin Exons 8\u201350 Results in DMD Phenotype"],"prefix":"10.3390","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3346-3242","authenticated-orcid":false,"given":"Tatiana V.","family":"Egorova","sequence":"first","affiliation":[{"name":"Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"},{"name":"Marlin Biotech LLC, Sochi 354340, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivan I.","family":"Galkin","sequence":"additional","affiliation":[{"name":"Marlin Biotech LLC, Sochi 354340, Russia"},{"name":"Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"},{"name":"Belozersky Institute of Physico-Chemical Biology, Lomonosov Moscow State University, Moscow 119991, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Oleg A.","family":"Velyaev","sequence":"additional","affiliation":[{"name":"Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Svetlana G.","family":"Vassilieva","sequence":"additional","affiliation":[{"name":"Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irina M.","family":"Savchenko","sequence":"additional","affiliation":[{"name":"Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"},{"name":"Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vyacheslav A.","family":"Loginov","sequence":"additional","affiliation":[{"name":"Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marina A.","family":"Dzhenkova","sequence":"additional","affiliation":[{"name":"Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana S.","family":"Korshunova","sequence":"additional","affiliation":[{"name":"Core Facilities, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olga S.","family":"Kozlova","sequence":"additional","affiliation":[{"name":"Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dmitry N.","family":"Ivankov","sequence":"additional","affiliation":[{"name":"Center for Molecular and Cellular Biology, Skolkovo Institute of Science and Technology, Moscow 121205, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna V.","family":"Polikarpova","sequence":"additional","affiliation":[{"name":"Laboratory of Modeling and Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow 119334, Russia"},{"name":"Marlin Biotech LLC, Sochi 354340, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2023,5,23]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/0888-7543(88)90113-9","article-title":"An Explanation for the Phenotypic Differences between Patients Bearing Partial Deletions of the DMD Locus","volume":"2","author":"Monaco","year":"1988","journal-title":"Genomics"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1186\/s13023-021-01862-w","article-title":"The Clinical Course of Duchenne Muscular Dystrophy in the Corticosteroid Treatment Era: A Systematic Literature Review","volume":"16","author":"Szabo","year":"2021","journal-title":"Orphanet J. 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