{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T14:01:14Z","timestamp":1753884074459,"version":"3.41.2"},"reference-count":40,"publisher":"The Company of Biologists","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"abstract":"<jats:p>Limb-girdle muscular dystrophy-2F (LGMD-2F) is an incurable degenerative muscle disorder caused by a mutation in the sarcoglycan-\u03b4 (SG\u03b4) gene. The lack of SG\u03b4 results in the complete disruption of the sarcoglycan complex (SGC) in the skeletal and cardiac muscle within the larger dystrophin-glycoprotein complex (DGC). The long-term consequences of SG\u03b4 ablation on other members of the DGC are currently unknown. We produced mosaic mice through the injection of wild type (WT) embryonic stem cells (ESCs) into SG\u03b4 knockout (KO) blastocysts. ESC-SG\u03b4 was supplied to the sarcolemma of the 18-month old chimeric muscle, which resulted in the restoration of the SGC. Despite SGC rescue, and contrary to previous observations obtained with WT\/mdx chimeras (a mouse rescue paradigm for Duchenne muscular dystrophy), low levels of ESC incorporation were insufficient to produce histological corrections in SG\u03b4 KO skeletal muscle or heart. The inefficient process of ESC rescue was more evident in the SG\u03b4 KO diaphragm, which had reduced levels of dystrophin and no compensatory utrophin, and needed almost full WT ESC reconstitution for histological improvement. The results suggest that the SG\u03b4 KO mouse model of LGMD is not amenable to ESC treatment.<\/jats:p>","DOI":"10.1242\/jcs.100537","type":"journal-article","created":{"date-parts":[[2012,2,11]],"date-time":"2012-02-11T04:27:03Z","timestamp":1328934423000},"source":"Crossref","is-referenced-by-count":2,"title":["Dystrophin-compromised sarcoglycan-\u03b4 knockout diaphragm requires full wild type embryonic stem cell reconstitution for correction"],"prefix":"10.1242","author":[{"given":"Joseph M.","family":"Vitale","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joel S.","family":"Schneider","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Amanda J.","family":"Beck","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Qingshi","family":"Zhao","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Corey","family":"Chang","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Richard","family":"Gordan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer","family":"Michaels","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mantu","family":"Bhaumik","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Diego","family":"Fraidenraich","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"237","published-online":{"date-parts":[[2012,1,1]]},"reference":[{"key":"2021042522285009600_R1","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1083\/jcb.200106158","article-title":"In vivo requirement of the alpha-syntrophin PDZ domain for the sarcolemmal localization of nNOS and aquaporin-4","volume":"155","author":"Adams","year":"2001","journal-title":"J. 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