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U.S.A."],"published-print":{"date-parts":[[2018,5,15]]},"abstract":"<jats:title>Significance<\/jats:title>\n                  <jats:p>\n                    The development of small-molecule therapeutics that act by targeting defined DNA or RNA sequences associated with human disease remains a challenge. RG-7916, a small-molecule drug candidate for the treatment of spinal muscular atrophy (SMA), selectively regulates the alternative splicing (AS) of the\n                    <jats:italic>SMN2<\/jats:italic>\n                    gene. Herein, we show that SMN-C2 and -C3, close analogs of RG-7916, act by binding SMN2 pre-mRNA and thereby increasing the affinity of the RNA binding proteins far upstream element binding protein 1 (FUBP1) and KH-type splicing regulatory protein (KHSRP) to the SMN2 pre-mRNA complex. These results suggest that nucleic acid targeted small molecules may have untapped potential for modulating disease processes at the level of pre-mRNA splicing.\n                  <\/jats:p>","DOI":"10.1073\/pnas.1800260115","type":"journal-article","created":{"date-parts":[[2018,4,30]],"date-time":"2018-04-30T15:16:04Z","timestamp":1525101364000},"update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":91,"title":["Mechanistic studies of a small-molecule modulator of SMN2 splicing"],"prefix":"10.1073","volume":"115","author":[{"given":"Jingxin","family":"Wang","sequence":"first","affiliation":[{"name":"California Institute for Biomedical Research, La Jolla, CA 92037;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter G.","family":"Schultz","sequence":"additional","affiliation":[{"name":"California Institute for Biomedical Research, La Jolla, CA 92037;"},{"name":"Department of Chemistry, The Scripps Research Institute, La Jolla, CA 92037;"},{"name":"Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, CA 92037"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristen A.","family":"Johnson","sequence":"additional","affiliation":[{"name":"California Institute for Biomedical Research, La Jolla, CA 92037;"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"341","published-online":{"date-parts":[[2018,4,30]]},"reference":[{"key":"e_1_3_4_1_2","doi-asserted-by":"crossref","first-page":"2120","DOI":"10.1016\/S0140-6736(08)60921-6","article-title":"Spinal muscular atrophy","volume":"371","author":"Lunn MR","year":"2008","unstructured":"MR Lunn, CH Wang, Spinal muscular atrophy. 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