{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T02:36:07Z","timestamp":1792636567344,"version":"4.5.0"},"reference-count":41,"publisher":"Portland Press Ltd.","issue":"4","license":[{"start":{"date-parts":[[2019,9,18]],"date-time":"2019-09-18T00:00:00Z","timestamp":1568764800000},"content-version":"vor","delay-in-days":229,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["portlandpress.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2019,2,28]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Recent reports, including ours, have indicated that microRNA (miR)-33 located within the intron of sterol regulatory element binding protein (SREBP) 2 controls cholesterol homeostasis and can be a potential therapeutic target for the treatment of atherosclerosis. Here, we show that SPAST, which encodes a microtubule-severing protein called SPASTIN, was a novel target gene of miR-33 in human. Actually, the miR-33 binding site in the SPAST 3\u2032-UTR is conserved not in mice but in mid to large mammals, and it is impossible to clarify the role of miR-33 on SPAST in mice. We demonstrated that inhibition of miR-33a, a major form of miR-33 in human neurons, via locked nucleic acid (LNA)-anti-miR ameliorated the pathological phenotype in hereditary spastic paraplegia (HSP)-SPG4 patient induced pluripotent stem cell (iPSC)-derived cortical neurons. Thus, miR-33a can be a potential therapeutic target for the treatment of HSP-SPG4.<\/jats:p>","DOI":"10.1042\/cs20180980","type":"journal-article","created":{"date-parts":[[2019,2,18]],"date-time":"2019-02-18T20:45:16Z","timestamp":1550522716000},"page":"583-595","update-policy":"https:\/\/doi.org\/10.1042\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["<i>MiR-33a<\/i> is a therapeutic target in SPG4-related hereditary spastic paraplegia human neurons"],"prefix":"10.1042","volume":"133","author":[{"given":"Fumiko","family":"Nakazeki","sequence":"first","affiliation":[{"name":"Department of Cardiovascular Medicine, Graduate School of Medicine, Kyoto University, Kyoto, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Itaru","family":"Tsuge","sequence":"additional","affiliation":[{"name":"Center for iPS Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan"},{"name":"Department of Plastic and Reconstructive Surgery, Graduate 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