{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T19:36:10Z","timestamp":1790537770959,"version":"4.1.0"},"reference-count":110,"publisher":"Informa UK Limited","issue":"13","funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["NS055925, NS072259 and NS080294 to RNS and NS060926"],"award-info":[{"award-number":["NS055925, NS072259 and NS080294 to RNS and NS060926"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["www.tandfonline.com"],"crossmark-restriction":true},"short-container-title":["Future Med. Chem."],"published-print":{"date-parts":[[2015,9]]},"DOI":"10.4155\/fmc.15.101","type":"journal-article","created":{"date-parts":[[2015,9,18]],"date-time":"2015-09-18T14:52:11Z","timestamp":1442587931000},"page":"1793-1808","update-policy":"https:\/\/doi.org\/10.1080\/tandf_crossmark_01","source":"Crossref","is-referenced-by-count":57,"title":["Mechanistic Principles of Antisense Targets for the Treatment of Spinal Muscular Atrophy"],"prefix":"10.1080","volume":"7","author":[{"given":"Natalia N","family":"Singh","sequence":"first","affiliation":[{"name":"Department of Biomedical Sciences, College of Veterinary Medicine, Iowa State University, Ames, IA50011, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian M","family":"Lee","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences, College of Veterinary Medicine, Iowa State University, Ames, IA50011, USA"},{"name":"Center for Advanced Host Defenses, Immunobiotics &amp; Translational Comparative Medicine (CAHDIT), Iowa State University, Ames, IA50011, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine J","family":"DiDonato","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, IL60611, USA"},{"name":"Human Molecular Genetics Program, Ann &amp; Robert H. Lurie Children's Hospital of Chicago, Stanley Manne Research Institute, Chicago, IL60614, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ravindra N","family":"Singh","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences, College of Veterinary Medicine, Iowa State University, Ames, IA50011, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"301","published-online":{"date-parts":[[2015,9,18]]},"reference":[{"key":"e_1_3_3_3_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073807305668"},{"key":"e_1_3_3_4_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneurol.2011.74"},{"key":"e_1_3_3_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.36251"},{"key":"e_1_3_3_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/ahg.12031"},{"key":"e_1_3_3_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90460-3"},{"key":"e_1_3_3_8_1","doi-asserted-by":"publisher","DOI":"10.1086\/515465"},{"key":"e_1_3_3_9_1","doi-asserted-by":"publisher","DOI":"10.4155\/fmc.14.63"},{"key":"e_1_3_3_10_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.11.6307"},{"key":"e_1_3_3_11_1","doi-asserted-by":"publisher","DOI":"10.1038\/ng0598-63"},{"key":"e_1_3_3_12_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddi078"},{"key":"e_1_3_3_13_1","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.01262-08"},{"key":"e_1_3_3_14_1","doi-asserted-by":"publisher","DOI":"10.1101\/gad.1884910"},{"key":"e_1_3_3_15_1","doi-asserted-by":"publisher","DOI":"10.1086\/338627"},{"key":"e_1_3_3_16_1","doi-asserted-by":"publisher","DOI":"10.1086\/302160"},{"key":"e_1_3_3_17_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00439-006-0156-7"},{"key":"e_1_3_3_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2007.02.006"},{"key":"e_1_3_3_19_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.94.18.9920"},{"key":"e_1_3_3_20_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0015887"},{"key":"e_1_3_3_21_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11910-010-0095-5"},{"key":"e_1_3_3_22_1","doi-asserted-by":"publisher","DOI":"10.1242\/dmm.007245"},{"key":"e_1_3_3_23_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00439-012-1171-5"},{"key":"e_1_3_3_24_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddn156"},{"key":"e_1_3_3_25_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddm367"},{"key":"e_1_3_3_26_1","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.4434-08.2009"},{"key":"e_1_3_3_27_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0015457"},{"key":"e_1_3_3_28_1","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.4496-09.2010"},{"key":"e_1_3_3_29_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ydbio.2011.05.667"},{"key":"e_1_3_3_30_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.neuron.2010.12.032"},{"key":"e_1_3_3_31_1","doi-asserted-by":"publisher","DOI":"10.1097\/NEN.0b013e31821cbd8b"},{"key":"e_1_3_3_32_1","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.5775-11.2012"},{"key":"e_1_3_3_33_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddq300"},{"key":"e_1_3_3_34_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddq330"},{"key":"e_1_3_3_35_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddq329"},{"key":"e_1_3_3_36_1","doi-asserted-by":"publisher","DOI":"10.1038\/ejhg.2012.222"},{"key":"e_1_3_3_37_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23582"},{"key":"e_1_3_3_38_1","doi-asserted-by":"publisher","DOI":"10.1111\/joa.12083"},{"key":"e_1_3_3_39_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddi130"},{"key":"e_1_3_3_40_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddp352"},{"key":"e_1_3_3_41_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI46276"},{"key":"e_1_3_3_42_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddr288"},{"key":"e_1_3_3_43_1","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.00945-10"},{"key":"e_1_3_3_44_1","doi-asserted-by":"publisher","DOI":"10.1021\/jm200497t"},{"key":"e_1_3_3_45_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1476-5381.2012.01829.x"},{"key":"e_1_3_3_46_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajhg.2012.05.001"},{"key":"e_1_3_3_47_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.1155085"},{"key":"e_1_3_3_48_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/dds263"},{"key":"e_1_3_3_49_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0049595"},{"key":"e_1_3_3_50_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.1250127"},{"key":"e_1_3_3_51_1","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.201207087"},{"key":"e_1_3_3_52_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2012.225"},{"key":"e_1_3_3_53_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23995"},{"key":"e_1_3_3_54_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbadis.2013.08.005"},{"key":"e_1_3_3_55_1","doi-asserted-by":"publisher","DOI":"10.1097\/MOP.0b013e32836565ac"},{"key":"e_1_3_3_56_1","doi-asserted-by":"publisher","DOI":"10.1007\/s13311-014-0293-y"},{"key":"e_1_3_3_57_1","doi-asserted-by":"publisher","DOI":"10.1007\/s13311-015-0337-y"},{"key":"e_1_3_3_58_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M107632200"},{"key":"e_1_3_3_59_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M200851200"},{"key":"e_1_3_3_60_1","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.26.4.1333-1346.2006"},{"key":"e_1_3_3_61_1","doi-asserted-by":"publisher","DOI":"10.4161\/rna.6.3.8723"},{"key":"e_1_3_3_62_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkt609"},{"key":"e_1_3_3_63_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2013.276"},{"key":"e_1_3_3_64_1","doi-asserted-by":"publisher","DOI":"10.2478\/s13380-013-0109-2"},{"key":"e_1_3_3_65_1","unstructured":"ClinicalTrials Database\n           : NCT02193074. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02193074."},{"key":"e_1_3_3_66_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrd3459"},{"key":"e_1_3_3_67_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrd3625"},{"key":"e_1_3_3_68_1","doi-asserted-by":"publisher","DOI":"10.1002\/wrna.1158"},{"key":"e_1_3_3_69_1","doi-asserted-by":"publisher","DOI":"10.4161\/rna.8.4.16224"},{"key":"e_1_3_3_70_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0700343104"},{"key":"e_1_3_3_71_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkl1050"},{"key":"e_1_3_3_72_1","doi-asserted-by":"publisher","DOI":"10.1261\/rna.7580704"},{"key":"e_1_3_3_73_1","doi-asserted-by":"publisher","DOI":"10.1038\/nsb887"},{"key":"e_1_3_3_74_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0633863100"},{"key":"e_1_3_3_75_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbrc.2004.01.067"},{"key":"e_1_3_3_76_1","doi-asserted-by":"publisher","DOI":"10.1615\/CritRevEukaryotGeneExpr.v14.i4.30"},{"key":"e_1_3_3_77_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddq094"},{"key":"e_1_3_3_78_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.1254806"},{"key":"e_1_3_3_79_1","first-page":"525","volume-title":"The RNA World (2nd Ed)","author":"Burge CB","year":"1999","unstructured":"Burge CB , TuschlT, SharpPA. Splicing of precursors to mRNAs by the spliceosomes. In : The RNA World (2nd Ed). GestelandRFet al.Cold Spring Harbor Laboratory Press, Cold Spring Harbor, NY, USA, 525\u2013560 (1999)."},{"key":"e_1_3_3_80_1","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.24.24.10505-10514.2004"},{"key":"e_1_3_3_81_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pbio.0040021"},{"key":"e_1_3_3_82_1","doi-asserted-by":"publisher","DOI":"10.1261\/rna.1069408"},{"key":"e_1_3_3_83_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.tibs.2009.10.004"},{"key":"e_1_3_3_84_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pbio.0050073"},{"key":"e_1_3_3_85_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajhg.2008.01.014"},{"key":"e_1_3_3_86_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40262-014-0224-4"},{"key":"e_1_3_3_87_1","doi-asserted-by":"publisher","DOI":"10.1038\/nature10485"},{"key":"e_1_3_3_88_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddr600"},{"key":"e_1_3_3_89_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2012.211"},{"key":"e_1_3_3_90_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0062114"},{"key":"e_1_3_3_91_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinthera.2014.02.004"},{"key":"e_1_3_3_92_1","doi-asserted-by":"publisher","DOI":"10.1261\/rna.2154310"},{"key":"e_1_3_3_93_1","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.00945-10"},{"key":"e_1_3_3_94_1","doi-asserted-by":"publisher","DOI":"10.1111\/nyas.12727"},{"key":"e_1_3_3_95_1","doi-asserted-by":"publisher","DOI":"10.1016\/bs.mie.2014.10.051"},{"key":"e_1_3_3_96_1","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2014.23"},{"key":"e_1_3_3_97_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddu198"},{"key":"e_1_3_3_98_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2011.07.014"},{"key":"e_1_3_3_99_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2005.08.022"},{"key":"e_1_3_3_100_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0008204"},{"key":"e_1_3_3_101_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkr152"},{"key":"e_1_3_3_102_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.celrep.2014.08.051"},{"key":"e_1_3_3_103_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.mt.6300200"},{"key":"e_1_3_3_104_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddn382"},{"key":"e_1_3_3_105_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2010.06.010"},{"key":"e_1_3_3_106_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2006.01.012"},{"key":"e_1_3_3_107_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2008.067"},{"key":"e_1_3_3_108_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddp076"},{"key":"e_1_3_3_109_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2011.232"},{"key":"e_1_3_3_110_1","doi-asserted-by":"publisher","DOI":"10.1038\/nature11556"},{"key":"e_1_3_3_111_1","doi-asserted-by":"publisher","DOI":"10.1038\/nature12831"},{"key":"e_1_3_3_112_1","doi-asserted-by":"publisher","DOI":"10.1038\/507423a"}],"container-title":["Future Medicinal Chemistry"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.tandfonline.com\/doi\/pdf\/10.4155\/fmc.15.101","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,5,23]],"date-time":"2024-05-23T19:50:35Z","timestamp":1716493835000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.tandfonline.com\/doi\/full\/10.4155\/fmc.15.101"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015,9]]},"references-count":110,"journal-issue":{"issue":"13","published-print":{"date-parts":[[2015,9]]}},"alternative-id":["10.4155\/fmc.15.101"],"URL":"https:\/\/doi.org\/10.4155\/fmc.15.101","relation":{},"ISSN":["1756-8919","1756-8927"],"issn-type":[{"value":"1756-8919","type":"print"},{"value":"1756-8927","type":"electronic"}],"subject":[],"published":{"date-parts":[[2015,9]]},"assertion":[{"value":"The publishing and review policy for this title is described in its Aims & Scope.","order":1,"name":"peerreview_statement","label":"Peer Review Statement"},{"value":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=ifmc20","URL":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=ifmc20","order":2,"name":"aims_and_scope_url","label":"Aim & Scope"},{"value":"2015-09-18","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}