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These diseases are currently incurable, emphasizing the importance of effective treatment strategies and the necessity of animal models for drug screening and therapeutic verification. Using the CRISPR\/Cas9 genome editing technique, we generated and characterized \u03b4-sarcoglycan and \u03b2-sarcoglycan knockout zebrafish lines, which presented a progressive disease phenotype that worsened from a mild larval stage to distinct myopathic features in adulthood. By subjecting the knockout larvae to a viscous swimming medium, we were able to anticipate disease onset. The \u03b4-SG knockout line was further exploited to demonstrate that a \u03b4-SG missense mutant is a substrate for endoplasmic reticulum-associated degradation (ERAD), indicating premature degradation due to protein folding defects. In conclusion, our study underscores the utility of zebrafish in modeling sarcoglycanopathies through either gene knockout or future knock-in techniques. These novel zebrafish lines will not only enhance our understanding of the disease\u2019s pathogenic mechanisms, but will also serve as powerful tools for phenotype-based drug screening, ultimately contributing to the development of a cure for sarcoglycanopathies.<\/jats:p>","DOI":"10.3390\/ijms241612707","type":"journal-article","created":{"date-parts":[[2023,8,11]],"date-time":"2023-08-11T12:10:30Z","timestamp":1691755830000},"page":"12707","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Modeling Sarcoglycanopathy in Danio rerio"],"prefix":"10.3390","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0009-0008-3277-4242","authenticated-orcid":false,"given":"Francesco","family":"Dalla Barba","sequence":"first","affiliation":[{"name":"Department of Biomedical Sciences, University of Padova, Via U. Bassi 58\/b, 35131 Padova, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michela","family":"Soardi","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences, University of Padova, Via U. Bassi 58\/b, 35131 Padova, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leila","family":"Mouhib","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences, University of Padova, Via U. Bassi 58\/b, 35131 Padova, Italy"},{"name":"Randall Center for Cell and Molecular Biophysics, King\u2019s College London, London WC2R 2LS, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0651-7409","authenticated-orcid":false,"given":"Giovanni","family":"Risato","sequence":"additional","affiliation":[{"name":"Department of Biology, University of Padova, Via U. Bassi 58\/b, 35131 Padova, Italy"},{"name":"Department of Cardiac-Thoracic-Vascular Sciences and Public Health, University of Padova, Via Giustiniani, 2, 35128 Padova, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7054-5116","authenticated-orcid":false,"given":"Eylem Emek","family":"Aky\u00fcrek","sequence":"additional","affiliation":[{"name":"Department of Comparative Biomedicine and Food Science, University of Padova, Agripolis, Legnaro, 35020 Padova, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tyrone","family":"Lucon-Xiccato","sequence":"additional","affiliation":[{"name":"Department of Life Sciences and Biotechnology, University of Ferrara, Via Luigi Borsari 46, 44121 Ferrara, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8761-1730","authenticated-orcid":false,"given":"Martina","family":"Scano","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences, University of Padova, Via U. Bassi 58\/b, 35131 Padova, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1848-0677","authenticated-orcid":false,"given":"Alberto","family":"Benetollo","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences, University of Padova, Via U. Bassi 58\/b, 35131 Padova, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberta","family":"Sacchetto","sequence":"additional","affiliation":[{"name":"Department of Comparative Biomedicine and Food Science, University of Padova, Agripolis, Legnaro, 35020 Padova, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6505-446X","authenticated-orcid":false,"given":"Isabelle","family":"Richard","sequence":"additional","affiliation":[{"name":"Genethon, F-91002 Evry, France"},{"name":"INSERM, U951, INTEGRARE Research Unit, F-91002 Evry, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0803-8236","authenticated-orcid":false,"given":"Francesco","family":"Argenton","sequence":"additional","affiliation":[{"name":"Department of Biology, University of Padova, Via U. Bassi 58\/b, 35131 Padova, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0252-3107","authenticated-orcid":false,"given":"Cristiano","family":"Bertolucci","sequence":"additional","affiliation":[{"name":"Department of Life Sciences and Biotechnology, University of Ferrara, Via Luigi Borsari 46, 44121 Ferrara, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7742-339X","authenticated-orcid":false,"given":"Marcello","family":"Carotti","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences, University of Padova, Via U. Bassi 58\/b, 35131 Padova, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9908-2442","authenticated-orcid":false,"given":"Dorianna","family":"Sandon\u00e0","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences, University of Padova, Via U. 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