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About 95% of SMA cases are caused by the loss of both copies of the\n                    <jats:italic>SMN1<\/jats:italic>\n                    gene.\n                    <jats:italic>SMN2<\/jats:italic>\n                    is a nearly identical copy of\n                    <jats:italic>SMN1<\/jats:italic>\n                    , which expresses much less functional SMN protein.\n                    <jats:italic>SMN2<\/jats:italic>\n                    is unable to fully compensate for the loss of\n                    <jats:italic>SMN1<\/jats:italic>\n                    in motor neurons but does provide an excellent target for therapeutic intervention. Increased expression of functional full\u2010length SMN protein from the endogenous\n                    <jats:italic>SMN2<\/jats:italic>\n                    gene should lessen disease severity. We have developed and implemented a new high\u2010throughput screening assay to identify small molecules that increase the expression of full\u2010length SMN from a\n                    <jats:italic>SMN2<\/jats:italic>\n                    reporter gene. Here, we characterize two novel compounds that increased SMN protein levels in both reporter cells and SMA fibroblasts and show that one increases lifespan, motor function, and SMN protein levels in a severe mouse model of SMA.\n                  <\/jats:p>","DOI":"10.1002\/emmm.201202305","type":"journal-article","created":{"date-parts":[[2013,6,5]],"date-time":"2013-06-05T08:37:20Z","timestamp":1370421440000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":41,"title":["Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug\u2010like compounds"],"prefix":"10.1038","volume":"5","author":[{"given":"Jonathan J.","family":"Cherry","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erkan Y.","family":"Osman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew C.","family":"Evans","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sungwoon","family":"Choi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xuechao","family":"Xing","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gregory D.","family":"Cuny","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcie A.","family":"Glicksman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christian L.","family":"Lorson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elliot J.","family":"Androphy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2013,6,5]]},"reference":[{"key":"BFEMMM201202305_CR1","doi-asserted-by":"crossref","first-page":"2841","DOI":"10.1093\/hmg\/10.24.2841","volume":"10","author":"C Andreassi","year":"2001","unstructured":"Andreassi C, Jarecki J, Zhou J, Coovert DD, Monani UR, Chen X, Whitney M, Pollok B, Zhang M, Androphy E, et al (2001) Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients. 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