{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,10]],"date-time":"2025-11-10T21:07:41Z","timestamp":1762808861324,"version":"3.40.5"},"reference-count":89,"publisher":"Bentham Science Publishers Ltd.","issue":"6","content-domain":{"domain":["eurekaselect.com"],"crossmark-restriction":true},"short-container-title":["CGT"],"published-print":{"date-parts":[[2020,4,25]]},"abstract":"<jats:sec>\n<jats:title\/>\n<jats:p>The Limb-Girdle Muscular Dystrophies (LGMD) are genetically heterogeneous disorders,\nresponsible for muscle wasting and severe form of dystrophies. Despite the critical developments in\nthe insight and information of pathomechanisms of limb-girdle muscular dystrophy, any definitive\ntreatments do not exist, and current strategies are only based on the improvement of the signs of disorder\nand to enhance the life quality without resolving an underlying cause. There is a crucial relationship\nbetween pharmacological therapy and different consequences; therefore, other treatment strategies\nwill be required. New approaches, such as gene replacement, gene transfer, exon skipping, siRNA\nknockdown, and anti-myostatin therapy, which can target specific cellular or molecular mechanism of\nLGMD, could be a promising avenue for the treatment. Recently, genome engineering strategies with\na focus on molecular tools such as CRISPR-Cas9 are used to different types of neuromuscular disorders\nand show the highest potential for clinical translation of these therapies. Thus, recent advancements\nand challenges in the field will be reviewed in this paper.<\/jats:p>\n<\/jats:sec>","DOI":"10.2174\/1566523220666200218113526","type":"journal-article","created":{"date-parts":[[2020,2,18]],"date-time":"2020-02-18T12:43:36Z","timestamp":1582029816000},"page":"386-394","update-policy":"https:\/\/doi.org\/10.2174\/bsp_crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Limb-girdle Muscular Dystrophy and Therapy: Insights into Cell and Gene-based Approaches"],"prefix":"10.2174","volume":"19","author":[{"given":"Forough","family":"Taheri","sequence":"first","affiliation":[{"name":"Shahrekord Branch, Islamic Azad University, Shahrekord, Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eskandar","family":"Taghizadeh","sequence":"additional","affiliation":[{"name":"Cellular and Molecular Research Center, Yasuj University of Medical Sciences, Yasuj, Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mohammad J.R.","family":"Pour","sequence":"additional","affiliation":[{"name":"Department of Biology, Faculty of Sciences, Mashhad-Branch, Islamic Azad University, Mashhad, Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Daryoush","family":"Rostami","sequence":"additional","affiliation":[{"name":"Department of School Allied, Zabol University of Medical Sciences, Zabol, Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pedram G.","family":"Renani","sequence":"additional","affiliation":[{"name":"Shahrekord Branch, Islamic Azad University, Shahrekord, Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Azam","family":"Rastgar-Moghadam","sequence":"additional","affiliation":[{"name":"Department of Genetics, Tehran Medical Sciences Branch, Islamic Azad University, Tehran, Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1378-118X","authenticated-orcid":true,"given":"Seyed M.G.","family":"Hayat","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, School of Medicine, Shahid Sadoughi University of Medical Sciences, Yazd, Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"965","reference":[{"key":"ref=1","doi-asserted-by":"publisher","first-page":"169","DOI":"10.1093\/brain\/77.2.169","volume":"77","author":"Walton J.N.","year":"1954","unstructured":"Walton J.N.; Nattrass F.J.; On the classification, natural history and treatment of the myopathies. 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