{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T19:48:40Z","timestamp":1791316120402,"version":"4.3.1"},"reference-count":101,"publisher":"SAGE Publications","issue":"5","license":[{"start":{"date-parts":[[2013,5,1]],"date-time":"2013-05-01T00:00:00Z","timestamp":1367366400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/www.liebertpub.com\/nv\/resources-tools\/text-and-data-mining-policy\/121\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2013,5]]},"DOI":"10.1089\/hum.2012.225","type":"journal-article","created":{"date-parts":[[2013,4,2]],"date-time":"2013-04-02T07:48:25Z","timestamp":1364888905000},"page":"489-498","source":"Crossref","is-referenced-by-count":81,"title":["Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy"],"prefix":"10.1177","volume":"24","author":[{"given":"Paul N.","family":"Porensky","sequence":"first","affiliation":[{"name":"Department of Molecular and Cellular Biochemistry, The Ohio State University, Columbus, OH 43210."},{"name":"Department of Neurological Surgery, The Ohio State University Medical Center, Columbus, OH 43210."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arthur H.M.","family":"Burghes","sequence":"additional","affiliation":[{"name":"Department of Neurological Surgery, The Ohio State University Medical Center, Columbus, OH 43210."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","reference":[{"key":"B1","doi-asserted-by":"publisher","DOI":"10.1177\/08830738050200110301"},{"key":"B2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2009.01.071"},{"key":"B3","doi-asserted-by":"publisher","DOI":"10.1097\/PHM.0b013e31804b1d66"},{"key":"B4","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddp076"},{"key":"B5","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pgen.1000773"},{"key":"B6","doi-asserted-by":"publisher","DOI":"10.2741\/3670"},{"key":"B7","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddq300"},{"key":"B8","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2011.157"},{"key":"B9","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2011.09.007"},{"key":"B10","doi-asserted-by":"publisher","DOI":"10.1038\/nrn2670"},{"key":"B11","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.01262-08"},{"key":"B12","doi-asserted-by":"publisher","DOI":"10.1038\/nsb887"},{"key":"B13","doi-asserted-by":"publisher","DOI":"10.1038\/nrg775"},{"key":"B14","doi-asserted-by":"publisher","DOI":"10.1086\/498853"},{"key":"B15","first-page":"S36.002","volume-title":"Annual Academy of Neurology meeting abstract","author":"Chirboga C.","year":"2013"},{"key":"B16","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.152.5.1107"},{"key":"B17","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/6.8.1205"},{"key":"B18","doi-asserted-by":"publisher","DOI":"10.1006\/nbdi.1996.0010"},{"key":"B19","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2008.067"},{"key":"B20","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddq514"},{"key":"B21","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2012.09.006"},{"key":"B22","doi-asserted-by":"publisher","DOI":"10.1159\/000006968"},{"key":"B23","doi-asserted-by":"publisher","DOI":"10.1038\/nbt.1610"},{"key":"B24","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/9.5.849"},{"key":"B25","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0000921"},{"key":"B26","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddm379"},{"key":"B27","doi-asserted-by":"publisher","DOI":"10.1186\/1472-6750-6-5"},{"key":"B28","doi-asserted-by":"publisher","DOI":"10.1006\/bbrc.1995.2135"},{"key":"B29","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.5775-11.2012"},{"key":"B30","doi-asserted-by":"publisher","DOI":"10.1016\/j.braindev.2005.02.009"},{"key":"B31","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddq330"},{"key":"B32","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.160181697"},{"key":"B33","doi-asserted-by":"publisher","DOI":"10.1038\/71709"},{"key":"B34","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajhg.2008.01.014"},{"key":"B35","doi-asserted-by":"publisher","DOI":"10.1101\/gad.1941310"},{"key":"B36","doi-asserted-by":"publisher","DOI":"10.1038\/nature10485"},{"key":"B37","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddi467"},{"key":"B38","doi-asserted-by":"publisher","DOI":"10.1038\/ng1207"},{"key":"B39","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0700343104"},{"key":"B40","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddi078"},{"key":"B41","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddr275"},{"key":"B42","doi-asserted-by":"publisher","DOI":"10.1038\/ng0797-265"},{"key":"B43","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M107632200"},{"key":"B44","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/9.2.259"},{"key":"B45","doi-asserted-by":"publisher","DOI":"10.1038\/ng0598-63"},{"key":"B46","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.11.6307"},{"key":"B47","doi-asserted-by":"publisher","DOI":"10.1002\/dvdy.21642"},{"key":"B48","doi-asserted-by":"publisher","DOI":"10.1172\/JCI57291"},{"key":"B49","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2005.08.022"},{"key":"B50","doi-asserted-by":"publisher","DOI":"10.1038\/sj.mt.6300200"},{"key":"B51","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.0204-12.2012"},{"key":"B52","doi-asserted-by":"publisher","DOI":"10.1261\/rna.1273209"},{"key":"B53","doi-asserted-by":"publisher","DOI":"10.1086\/515465"},{"key":"B54","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddn189"},{"key":"B55","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.200303168"},{"key":"B56","doi-asserted-by":"publisher","DOI":"10.1016\/j.neuron.2010.12.032"},{"key":"B57","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddn382"},{"key":"B58","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0062114"},{"key":"B59","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M200851200"},{"key":"B60","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M209271200"},{"key":"B61","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/8.7.1177"},{"key":"B62","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/9.16.2451"},{"key":"B63","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/9.3.333"},{"key":"B64","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2011.232"},{"key":"B65","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.2208-10.2010"},{"key":"B66","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/5.11.1727"},{"key":"B67","doi-asserted-by":"publisher","DOI":"10.1086\/302160"},{"key":"B68","doi-asserted-by":"publisher","DOI":"10.1172\/JCI41615"},{"key":"B69","first-page":"72ra18","volume":"3","author":"Passini M.A.","year":"2011","journal-title":"Med."},{"key":"B70","doi-asserted-by":"publisher","DOI":"10.1136\/jmg.15.6.409"},{"key":"B71","doi-asserted-by":"publisher","DOI":"10.1016\/S0092-8674(00)81632-3"},{"key":"B72","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-008-0929-0"},{"key":"B73","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddr600"},{"key":"B74","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajhg.2009.08.002"},{"key":"B75","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.33474"},{"key":"B76","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2011.10.012"},{"key":"B77","doi-asserted-by":"publisher","DOI":"10.1136\/adc.45.239.33"},{"key":"B78","doi-asserted-by":"publisher","DOI":"10.1007\/s004390100473"},{"key":"B79","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddn426"},{"key":"B80","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.200304128"},{"key":"B81","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.06077-11"},{"key":"B82","doi-asserted-by":"publisher","DOI":"10.1177\/0883073807305667"},{"key":"B83","doi-asserted-by":"publisher","DOI":"10.4161\/rna.4.1.4534"},{"key":"B84","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.94.18.9920"},{"key":"B85","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddq329"},{"key":"B86","doi-asserted-by":"publisher","DOI":"10.1016\/j.yjmcc.2012.01.005"},{"key":"B87","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.26.4.1333-1346.2006"},{"key":"B88","doi-asserted-by":"publisher","DOI":"10.1261\/rna.7580704"},{"key":"B89","doi-asserted-by":"publisher","DOI":"10.1615\/CritRevEukaryotGeneExpr.v14.i4.30"},{"key":"B90","doi-asserted-by":"publisher","DOI":"10.4161\/rna.6.3.8723"},{"key":"B91","doi-asserted-by":"publisher","DOI":"10.4161\/rna.4.1.4535"},{"key":"B92","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0633863100"},{"key":"B93","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddp253"},{"key":"B94","doi-asserted-by":"publisher","DOI":"10.1002\/ana.20473"},{"key":"B95","first-page":"35ra42","volume":"2","author":"Valori C.F.","year":"2010","journal-title":"Med."},{"key":"B96","doi-asserted-by":"publisher","DOI":"10.1002\/humu.21173"},{"key":"B97","doi-asserted-by":"publisher","DOI":"10.1016\/S0002-9440(10)63428-1"},{"key":"B98","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.0950-09.2009"},{"key":"B99","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddp157"},{"key":"B100","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.1995.00540290108025"},{"key":"B101","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2012.211"}],"container-title":["Human Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.liebertpub.com\/doi\/full-xml\/10.1089\/hum.2012.225","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/www.liebertpub.com\/doi\/pdf\/10.1089\/hum.2012.225","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T07:55:23Z","timestamp":1777449323000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/full\/10.1089\/hum.2012.225"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,5]]},"references-count":101,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2013,5]]}},"alternative-id":["10.1089\/hum.2012.225"],"URL":"https:\/\/doi.org\/10.1089\/hum.2012.225","relation":{},"ISSN":["1043-0342","1557-7422"],"issn-type":[{"value":"1043-0342","type":"print"},{"value":"1557-7422","type":"electronic"}],"subject":[],"published":{"date-parts":[[2013,5]]}}}