{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T03:08:51Z","timestamp":1791169731044,"version":"4.1.0"},"reference-count":38,"publisher":"National Academy of Sciences","issue":"17","content-domain":{"domain":["www.pnas.org"],"crossmark-restriction":true},"short-container-title":["Proc. Natl. Acad. Sci. U.S.A."],"published-print":{"date-parts":[[2001,8,14]]},"abstract":"<jats:p>\n                    Spinal muscular atrophy (SMA) is an autosomal recessive disease\n characterized by degeneration of the anterior horn cells of the spinal\n cord, leading to muscular paralysis with muscular atrophy. No effective\n treatment of this disorder is presently available. Studies of the\n correlation between disease severity and the amount of survival motor\n neuron (SMN) protein have shown an inverse relationship. We report that\n sodium butyrate effectively increases the amount of exon 7-containing\n SMN protein in SMA lymphoid cell lines by changing the alternative\n splicing pattern of exon 7 in the\n                    <jats:italic>SMN<\/jats:italic>\n                    2 gene.\n                    <jats:italic>In\n vivo<\/jats:italic>\n                    , sodium butyrate treatment of SMA-like mice resulted in\n increased expression of SMN protein in motor neurons of the spinal cord\n and resulted in significant improvement of SMA clinical symptoms. Oral\n administration of sodium butyrate to intercrosses of heterozygous\n pregnant knockout-transgenic SMA-like mice decreased the birth rate of\n severe types of SMA-like mice, and SMA symptoms were ameliorated for\n all three types of SMA-like mice. These results suggest that sodium\n butyrate may be an effective drug for the treatment of human SMA\n patients.\n                  <\/jats:p>","DOI":"10.1073\/pnas.171105098","type":"journal-article","created":{"date-parts":[[2002,7,26]],"date-time":"2002-07-26T10:34:10Z","timestamp":1027679650000},"page":"9808-9813","update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":328,"title":["Treatment of spinal muscular atrophy by sodium butyrate"],"prefix":"10.1073","volume":"98","author":[{"given":"Jan-Gowth","family":"Chang","sequence":"first","affiliation":[{"name":"Department of Medical Research, China Medical College Hospital,\r Taichung 404, Taiwan; Institute of Molecular Biology,\r Academia Sinica, Taipei 115, Taiwan; and Departments of\r Pediatrics and Clinical Laboratory, Kaohsiung Medical University,\r Kaohsiung 807, Taiwan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hsiu-Mei","family":"Hsieh-Li","sequence":"additional","affiliation":[{"name":"Department of Medical Research, China Medical College Hospital,\r Taichung 404, Taiwan; Institute of Molecular Biology,\r Academia Sinica, Taipei 115, Taiwan; and Departments of\r Pediatrics and Clinical Laboratory, Kaohsiung Medical University,\r Kaohsiung 807, Taiwan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuh-Jyh","family":"Jong","sequence":"additional","affiliation":[{"name":"Department of Medical Research, China Medical College Hospital,\r Taichung 404, Taiwan; Institute of Molecular Biology,\r Academia Sinica, Taipei 115, Taiwan; and Departments of\r Pediatrics and Clinical Laboratory, Kaohsiung Medical University,\r Kaohsiung 807, Taiwan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nancy M.","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Medical Research, China Medical College Hospital,\r Taichung 404, Taiwan; Institute of Molecular Biology,\r Academia Sinica, Taipei 115, Taiwan; and Departments of\r Pediatrics and Clinical Laboratory, Kaohsiung Medical University,\r Kaohsiung 807, Taiwan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chang-Hai","family":"Tsai","sequence":"additional","affiliation":[{"name":"Department of Medical Research, China Medical College Hospital,\r Taichung 404, Taiwan; Institute of Molecular Biology,\r Academia Sinica, Taipei 115, Taiwan; and Departments of\r Pediatrics and Clinical Laboratory, Kaohsiung Medical University,\r Kaohsiung 807, Taiwan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hung","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Medical Research, China Medical College Hospital,\r Taichung 404, Taiwan; Institute of Molecular Biology,\r Academia Sinica, Taipei 115, Taiwan; and Departments of\r Pediatrics and Clinical Laboratory, 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