{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T03:54:00Z","timestamp":1790394840575,"version":"4.1.0"},"reference-count":61,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2019,12,31]],"date-time":"2019-12-31T00:00:00Z","timestamp":1577750400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"SMA Europe, FundAME","award":["19243"],"award-info":[{"award-number":["19243"]}]},{"name":"AFM-Telethon","award":["22346"],"award-info":[{"award-number":["22346"]}]}],"content-domain":{"domain":["faseb.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["The FASEB Journal"],"published-print":{"date-parts":[[2020,2]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Spinal muscular atrophy is a rare and fatal neuromuscular disorder caused by the loss of alpha motor neurons. The affected individuals have mutated the ubiquitously expressed\n                    <jats:italic>SMN1<\/jats:italic>\n                    gene resulting in the loss or reduction in the survival motor neuron (SMN) protein levels. However, an almost identical paralog exists in humans:\n                    <jats:italic>SMN2<\/jats:italic>\n                    . Pharmacological activation of\n                    <jats:italic>SMN2<\/jats:italic>\n                    exon 7 inclusion by small molecules or modified antisense oligonucleotides is a valid approach to treat SMA. Here we describe an in vivo\n                    <jats:italic>SMN2<\/jats:italic>\n                    minigene reporter system in\n                    <jats:italic>Drosophila<\/jats:italic>\n                    motor neurons that serves as a cost\u2010effective, feasible, and stringent primary screening model for identifying chemicals capable of crossing the conserved\n                    <jats:italic>Drosophila<\/jats:italic>\n                    blood\u2010brain barrier and modulating exon 7 inclusion. The model was used for the screening of 1100 drugs from the Prestwick Chemical Library, resulting in 2.45% hit rate. The most promising candidate drugs were validated in patient\u2010derived fibroblasts where they proved to increase SMN protein levels. Among them, moxifloxacin modulated\n                    <jats:italic>SMN2<\/jats:italic>\n                    splicing by promoting exon 7 inclusion. The recovery of SMN protein levels was confirmed by increased colocalization of nuclear gems with Cajal Bodies. Thus, a\n                    <jats:italic>Drosophila<\/jats:italic>\n                    \u2010based drug screen allowed the discovery of an FDA\u2010approved small molecule with the potential to become a novel therapy for SMA.\n                  <\/jats:p>","DOI":"10.1096\/fj.201802554rrr","type":"journal-article","created":{"date-parts":[[2019,12,31]],"date-time":"2019-12-31T04:56:33Z","timestamp":1577768193000},"page":"3021-3036","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":16,"title":["<i>Drosophila SMN2<\/i>\n                    minigene reporter model identifies moxifloxacin as a candidate therapy for SMA"],"prefix":"10.1096","volume":"34","author":[{"given":"Piotr","family":"Konieczny","sequence":"first","affiliation":[{"name":"Interdisciplinary Research Structure for Biotechnology and Biomedicine (ERI BIOTECMED) University of Valencia Valencia Spain"},{"name":"Translational Genomics Group Incliva Health Research Institute Valencia Spain"},{"name":"Incliva\u2010CIPF Joint Unit Valencia Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rub\u00e9n","family":"Artero","sequence":"additional","affiliation":[{"name":"Interdisciplinary Research Structure for Biotechnology and Biomedicine (ERI BIOTECMED) University of Valencia Valencia Spain"},{"name":"Translational Genomics Group Incliva Health Research Institute Valencia Spain"},{"name":"Incliva\u2010CIPF Joint Unit Valencia 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