{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T09:45:57Z","timestamp":1791279957087,"version":"4.1.0"},"reference-count":37,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2003,10,22]],"date-time":"2003-10-22T00:00:00Z","timestamp":1066780800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Annals of Neurology"],"published-print":{"date-parts":[[2003,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Spinal muscular atrophy (SMA) is an inherited motor neuron disease caused by mutation of the telomeric copy of the survival motor neuron gene (<jats:italic>SMN1<\/jats:italic>). Although a centromeric copy of the survival motor neuron gene <jats:italic>(SMN2)<\/jats:italic> is retained in all patients with SMA, it differs from <jats:italic>SMN1<\/jats:italic> at a critical nucleotide such that the majority of <jats:italic>SMN2<\/jats:italic> transcripts lack exon 7 and encode an unstable, truncated protein. Here, we show that valproic acid increases levels of exon 7\u2013containing <jats:italic>SMN<\/jats:italic> transcript and SMN protein in type I SMA patient\u2013derived fibroblast cell lines. Valproic acid may increase SMN levels both by activating the <jats:italic>SMN<\/jats:italic> promoter and by preventing exon 7 skipping in <jats:italic>SMN<\/jats:italic> transcripts. Valproic acid and related compounds warrant further investigation as potential treatment for SMA. Ann Neurol 2003;54:647\u2013654<\/jats:p>","DOI":"10.1002\/ana.10743","type":"journal-article","created":{"date-parts":[[2003,10,24]],"date-time":"2003-10-24T08:10:06Z","timestamp":1066983006000},"page":"647-654","source":"Crossref","is-referenced-by-count":247,"title":["Valproic acid increases SMN levels in spinal muscular atrophy patient cells"],"prefix":"10.1002","volume":"54","author":[{"given":"Charlotte J.","family":"Sumner","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thanh N.","family":"Huynh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer A.","family":"Markowitz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J. 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