{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T11:54:18Z","timestamp":1792756458846,"version":"4.5.0"},"reference-count":168,"publisher":"Oxford University Press (OUP)","issue":"14","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Nucleic Acids Res"],"published-print":{"date-parts":[[2016,8,19]]},"DOI":"10.1093\/nar\/gkw533","type":"journal-article","created":{"date-parts":[[2016,6,10]],"date-time":"2016-06-10T22:45:42Z","timestamp":1465598742000},"page":"6549-6563","source":"Crossref","is-referenced-by-count":553,"title":["Splice-switching antisense oligonucleotides as therapeutic drugs"],"prefix":"10.1093","volume":"44","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3416-3572","authenticated-orcid":false,"given":"Mallory A.","family":"Havens","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michelle L.","family":"Hastings","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2016,6,10]]},"reference":[{"key":"2016081822022971000_44.14.6549.1","doi-asserted-by":"publisher","DOI":"10.1016\/j.tibs.2015.11.003"},{"key":"2016081822022971000_44.14.6549.2","doi-asserted-by":"publisher","DOI":"10.1126\/science.aac8159"},{"key":"2016081822022971000_44.14.6549.3","doi-asserted-by":"publisher","DOI":"10.1126\/science.aad6466"},{"key":"2016081822022971000_44.14.6549.4","doi-asserted-by":"publisher","DOI":"10.1126\/science.3890181"},{"key":"2016081822022971000_44.14.6549.5","doi-asserted-by":"publisher","DOI":"10.1101\/cshperspect.a003707"},{"key":"2016081822022971000_44.14.6549.6","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-biochem-060614-034316"},{"key":"2016081822022971000_44.14.6549.7","doi-asserted-by":"publisher","DOI":"10.1038\/ng.259"},{"key":"2016081822022971000_44.14.6549.8","doi-asserted-by":"publisher","DOI":"10.1261\/rna.876308"},{"key":"2016081822022971000_44.14.6549.9","doi-asserted-by":"publisher","DOI":"10.1038\/nature09000"},{"key":"2016081822022971000_44.14.6549.10","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-biochem-080111-092106"},{"key":"2016081822022971000_44.14.6549.11","doi-asserted-by":"publisher","DOI":"10.1016\/j.gde.2011.03.004"},{"key":"2016081822022971000_44.14.6549.12","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2004.11.039"},{"key":"2016081822022971000_44.14.6549.13","doi-asserted-by":"publisher","DOI":"10.1038\/nrm1645"},{"key":"2016081822022971000_44.14.6549.14","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1126\/science.1254806","article-title":"RNA splicing. The human splicing code reveals new insights into the genetic determinants of disease","volume":"347","author":"Xiong","year":"2015","journal-title":"Science"},{"key":"2016081822022971000_44.14.6549.15","doi-asserted-by":"publisher","DOI":"10.1038\/nrg2164"},{"key":"2016081822022971000_44.14.6549.16","doi-asserted-by":"publisher","DOI":"10.1093\/bioinformatics\/btu277"},{"key":"2016081822022971000_44.14.6549.17","doi-asserted-by":"publisher","DOI":"10.1126\/science.aaa8381"},{"key":"2016081822022971000_44.14.6549.18","doi-asserted-by":"publisher","DOI":"10.1126\/science.1230612"},{"key":"2016081822022971000_44.14.6549.19","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.201510032"},{"key":"2016081822022971000_44.14.6549.20","doi-asserted-by":"publisher","DOI":"10.1002\/wrna.1158"},{"key":"2016081822022971000_44.14.6549.21","doi-asserted-by":"publisher","DOI":"10.1186\/gb4150"},{"key":"2016081822022971000_44.14.6549.22","doi-asserted-by":"publisher","DOI":"10.1016\/S0167-4781(99)00150-5"},{"key":"2016081822022971000_44.14.6549.23","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-4939-1221-6_9"},{"key":"2016081822022971000_44.14.6549.24","doi-asserted-by":"publisher","DOI":"10.4155\/fmc.15.144"},{"key":"2016081822022971000_44.14.6549.25","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-61779-767-5_23"},{"key":"2016081822022971000_44.14.6549.26","doi-asserted-by":"publisher","DOI":"10.1016\/j.addr.2015.03.008"},{"key":"2016081822022971000_44.14.6549.27","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/16.8.3209"},{"key":"2016081822022971000_44.14.6549.28","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2014.0506"},{"key":"2016081822022971000_44.14.6549.29","doi-asserted-by":"publisher","DOI":"10.1517\/17425250902877680"},{"key":"2016081822022971000_44.14.6549.30","doi-asserted-by":"publisher","DOI":"10.1039\/c1cs15048k"},{"key":"2016081822022971000_44.14.6549.31","doi-asserted-by":"publisher","DOI":"10.1002\/ana.21627"},{"key":"2016081822022971000_44.14.6549.32","doi-asserted-by":"publisher","DOI":"10.1124\/jpet.113.212407"},{"key":"2016081822022971000_44.14.6549.33","doi-asserted-by":"crossref","unstructured":"Juliano R.L. The delivery of therapeutic oligonucleotides. Nucleic Acids Res. 2016. doi:10.1093\/nar\/gkw236.","DOI":"10.1093\/nar\/gkw236"},{"key":"2016081822022971000_44.14.6549.34","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-61779-767-5_25"},{"key":"2016081822022971000_44.14.6549.35","doi-asserted-by":"publisher","DOI":"10.1016\/j.addr.2015.01.008"},{"key":"2016081822022971000_44.14.6549.36","doi-asserted-by":"publisher","DOI":"10.1038\/nature10485"},{"key":"2016081822022971000_44.14.6549.37","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2013.0443"},{"key":"2016081822022971000_44.14.6549.38","doi-asserted-by":"publisher","DOI":"10.1172\/JCI25424"},{"key":"2016081822022971000_44.14.6549.39","doi-asserted-by":"publisher","DOI":"10.1016\/j.molmed.2012.09.001"},{"key":"2016081822022971000_44.14.6549.40","doi-asserted-by":"publisher","DOI":"10.1038\/nm.3106"},{"key":"2016081822022971000_44.14.6549.41","doi-asserted-by":"publisher","DOI":"10.1101\/gad.1941310"},{"key":"2016081822022971000_44.14.6549.42","doi-asserted-by":"publisher","DOI":"10.15252\/emmm.201505846"},{"key":"2016081822022971000_44.14.6549.43","doi-asserted-by":"publisher","DOI":"10.1177\/0883073815627882"},{"key":"2016081822022971000_44.14.6549.44","doi-asserted-by":"publisher","DOI":"10.1016\/j.pharmthera.2014.05.009"},{"key":"2016081822022971000_44.14.6549.45","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkr089"},{"key":"2016081822022971000_44.14.6549.46","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1002\/ana.24555","article-title":"Longitudinal effect of eteplirsen vs. historical control on ambulation in DMD","volume":"79","author":"Mendell","year":"2015","journal-title":"Ann. Neurol."},{"key":"2016081822022971000_44.14.6549.47","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(11)60756-3"},{"key":"2016081822022971000_44.14.6549.48","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/28.2.582"},{"key":"2016081822022971000_44.14.6549.49","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2012.30"},{"key":"2016081822022971000_44.14.6549.50","doi-asserted-by":"publisher","DOI":"10.3390\/molecules14031304"},{"key":"2016081822022971000_44.14.6549.51","doi-asserted-by":"publisher","DOI":"10.2174\/156652312800840612"},{"key":"2016081822022971000_44.14.6549.52","doi-asserted-by":"publisher","DOI":"10.1038\/ncomms10981"},{"key":"2016081822022971000_44.14.6549.53","doi-asserted-by":"crossref","first-page":"2242","DOI":"10.1038\/srep02242","article-title":"Ultrasound-enhanced delivery of morpholino with Bubble liposomes ameliorates the myotonia of myotonic dystrophy model mice","volume":"3","author":"Koebis","year":"2013","journal-title":"Sci. Rep."},{"key":"2016081822022971000_44.14.6549.54","doi-asserted-by":"publisher","DOI":"10.1021\/mp4004755"},{"key":"2016081822022971000_44.14.6549.55","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0406700102"},{"key":"2016081822022971000_44.14.6549.56","doi-asserted-by":"crossref","first-page":"ddw118","DOI":"10.1093\/hmg\/ddw118","article-title":"In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery","volume":"2016","author":"Garanto","year":"2016","journal-title":"Hum. Mol. Genet."},{"key":"2016081822022971000_44.14.6549.57","doi-asserted-by":"crossref","first-page":"2296","DOI":"10.1016\/j.bbamem.2010.02.012","article-title":"Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy","volume":"1798","author":"Moulton","year":"2010","journal-title":"Biochim. Biophys. Acta"},{"key":"2016081822022971000_44.14.6549.58","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gks710"},{"key":"2016081822022971000_44.14.6549.59","doi-asserted-by":"publisher","DOI":"10.1016\/j.molcel.2011.08.009"},{"key":"2016081822022971000_44.14.6549.60","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.90.18.8673"},{"key":"2016081822022971000_44.14.6549.61","doi-asserted-by":"publisher","DOI":"10.2741\/4367"},{"key":"2016081822022971000_44.14.6549.62","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2013.0461"},{"key":"2016081822022971000_44.14.6549.63","doi-asserted-by":"publisher","DOI":"10.1016\/j.coph.2015.07.005"},{"key":"2016081822022971000_44.14.6549.64","doi-asserted-by":"crossref","first-page":"R18","DOI":"10.1093\/hmg\/ddv476","article-title":"Gene-targeting pharmaceuticals for single-gene disorders","volume":"25","author":"Beaudet","year":"2015","journal-title":"Hum. Mol. Genet."},{"key":"2016081822022971000_44.14.6549.65","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2013.234"},{"key":"2016081822022971000_44.14.6549.66","first-page":"245","article-title":"Splicing modulation therapy in the treatment of genetic diseases","volume":"7","author":"Arechavala-Gomeza","year":"2014","journal-title":"Appl. Clin. Genet."},{"key":"2016081822022971000_44.14.6549.67","doi-asserted-by":"publisher","DOI":"10.1097\/WCO.0000000000000235"},{"key":"2016081822022971000_44.14.6549.68","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2013.05.008"},{"key":"2016081822022971000_44.14.6549.69","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2014-2044"},{"key":"2016081822022971000_44.14.6549.70","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23810"},{"key":"2016081822022971000_44.14.6549.71","doi-asserted-by":"publisher","DOI":"10.1038\/sj.embor.7400221"},{"key":"2016081822022971000_44.14.6549.72","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1097\/00001573-199705000-00015","article-title":"Dystrophies and heart disease","volume":"12","author":"Cox","year":"1997","journal-title":"Curr. Opin. Cardiol."},{"key":"2016081822022971000_44.14.6549.73","doi-asserted-by":"publisher","DOI":"10.1016\/0888-7543(88)90113-9"},{"key":"2016081822022971000_44.14.6549.74","first-page":"498","article-title":"The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion","volume":"45","author":"Koenig","year":"1989","journal-title":"Am. J. Hum. Genet."},{"key":"2016081822022971000_44.14.6549.75","doi-asserted-by":"publisher","DOI":"10.1086\/381039"},{"key":"2016081822022971000_44.14.6549.76","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2013.0451"},{"key":"2016081822022971000_44.14.6549.77","doi-asserted-by":"publisher","DOI":"10.1172\/JCI117693"},{"key":"2016081822022971000_44.14.6549.78","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/7.7.1083"},{"key":"2016081822022971000_44.14.6549.79","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/10.15.1547"},{"key":"2016081822022971000_44.14.6549.80","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.98.1.42"},{"key":"2016081822022971000_44.14.6549.81","doi-asserted-by":"publisher","DOI":"10.1016\/j.coph.2005.06.001"},{"key":"2016081822022971000_44.14.6549.82","doi-asserted-by":"publisher","DOI":"10.1002\/jgm.1288"},{"key":"2016081822022971000_44.14.6549.83","doi-asserted-by":"publisher","DOI":"10.1038\/nm1345"},{"key":"2016081822022971000_44.14.6549.84","doi-asserted-by":"crossref","first-page":"e44","DOI":"10.1038\/mtna.2012.38","article-title":"Long-term Exon Skipping Studies With 2\u2032-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models","volume":"1","author":"Tanganyika-de\u00a0Winter","year":"2012","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"2016081822022971000_44.14.6549.85","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1204638109"},{"key":"2016081822022971000_44.14.6549.86","doi-asserted-by":"publisher","DOI":"10.1203\/01.pdr.0000215047.51278.7c"},{"key":"2016081822022971000_44.14.6549.87","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2012.234"},{"key":"2016081822022971000_44.14.6549.88","doi-asserted-by":"publisher","DOI":"10.1089\/humc.2015.2528"},{"key":"2016081822022971000_44.14.6549.89","doi-asserted-by":"publisher","DOI":"10.1038\/nrg1180"},{"key":"2016081822022971000_44.14.6549.90","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.142302299"},{"key":"2016081822022971000_44.14.6549.91","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2006.061"},{"key":"2016081822022971000_44.14.6549.92","doi-asserted-by":"publisher","DOI":"10.1177\/1091581809359206"},{"key":"2016081822022971000_44.14.6549.93","doi-asserted-by":"publisher","DOI":"10.1177\/1091581811403504"},{"key":"2016081822022971000_44.14.6549.94","doi-asserted-by":"publisher","DOI":"10.1177\/1091581811403505"},{"key":"2016081822022971000_44.14.6549.95","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70211-X"},{"key":"2016081822022971000_44.14.6549.96","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23982"},{"key":"2016081822022971000_44.14.6549.97","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa073108"},{"key":"2016081822022971000_44.14.6549.98","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(14)70195-4"},{"key":"2016081822022971000_44.14.6549.99","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1011367"},{"key":"2016081822022971000_44.14.6549.100","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2014.6"},{"key":"2016081822022971000_44.14.6549.101","doi-asserted-by":"publisher","DOI":"10.2174\/156652312800840621"},{"key":"2016081822022971000_44.14.6549.102","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2015.10.007"},{"key":"2016081822022971000_44.14.6549.103","doi-asserted-by":"publisher","DOI":"10.1016\/0736-5748(91)90022-E"},{"key":"2016081822022971000_44.14.6549.104","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2014.53"},{"key":"2016081822022971000_44.14.6549.105","doi-asserted-by":"publisher","DOI":"10.1242\/dmm.001008"},{"key":"2016081822022971000_44.14.6549.106","doi-asserted-by":"publisher","DOI":"10.1016\/j.ncl.2015.07.004"},{"key":"2016081822022971000_44.14.6549.107","doi-asserted-by":"crossref","first-page":"1608","DOI":"10.1002\/ajmg.a.33474","article-title":"Newborn and carrier screening for spinal muscular atrophy","volume":"152","author":"Prior","year":"2010","journal-title":"Am. J. Med. Genet. A"},{"key":"2016081822022971000_44.14.6549.108","doi-asserted-by":"publisher","DOI":"10.1038\/ejhg.2011.134"},{"key":"2016081822022971000_44.14.6549.109","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1186\/s12881-015-0246-2","article-title":"Validation of a high resolution NGS method for detecting spinal muscular atrophy carriers among phase 3 participants in the 1000 Genomes Project","volume":"16","author":"Larson","year":"2015","journal-title":"BMC Med. Genet."},{"key":"2016081822022971000_44.14.6549.110","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"2016081822022971000_44.14.6549.111","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2012.05.067"},{"key":"2016081822022971000_44.14.6549.112","doi-asserted-by":"publisher","DOI":"10.1111\/j.1399-0004.2009.01200.x"},{"key":"2016081822022971000_44.14.6549.113","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.11.6307"},{"key":"2016081822022971000_44.14.6549.114","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/8.7.1177"},{"key":"2016081822022971000_44.14.6549.115","doi-asserted-by":"publisher","DOI":"10.1038\/ng854"},{"key":"2016081822022971000_44.14.6549.116","doi-asserted-by":"publisher","DOI":"10.1086\/498853"},{"key":"2016081822022971000_44.14.6549.117","doi-asserted-by":"publisher","DOI":"10.1038\/ng1207"},{"key":"2016081822022971000_44.14.6549.118","doi-asserted-by":"publisher","DOI":"10.1101\/gad.1884910"},{"key":"2016081822022971000_44.14.6549.119","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/9.2.259"},{"key":"2016081822022971000_44.14.6549.120","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90460-3"},{"key":"2016081822022971000_44.14.6549.121","doi-asserted-by":"publisher","DOI":"10.1038\/3030"},{"key":"2016081822022971000_44.14.6549.122","doi-asserted-by":"publisher","DOI":"10.1038\/ng0797-265"},{"key":"2016081822022971000_44.14.6549.123","doi-asserted-by":"publisher","DOI":"10.1086\/515465"},{"key":"2016081822022971000_44.14.6549.124","doi-asserted-by":"publisher","DOI":"10.1007\/s00439-006-0156-7"},{"key":"2016081822022971000_44.14.6549.125","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(12)70061-3"},{"key":"2016081822022971000_44.14.6549.126","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M107632200"},{"key":"2016081822022971000_44.14.6549.127","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M200851200"},{"key":"2016081822022971000_44.14.6549.128","doi-asserted-by":"publisher","DOI":"10.1038\/nsb887"},{"key":"2016081822022971000_44.14.6549.129","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0633863100"},{"key":"2016081822022971000_44.14.6549.130","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.0417-15.2015"},{"key":"2016081822022971000_44.14.6549.131","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0062114"},{"key":"2016081822022971000_44.14.6549.132","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2012.211"},{"key":"2016081822022971000_44.14.6549.133","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddu198"},{"key":"2016081822022971000_44.14.6549.134","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2014.23"},{"key":"2016081822022971000_44.14.6549.135","doi-asserted-by":"publisher","DOI":"10.4161\/rna.6.3.8723"},{"key":"2016081822022971000_44.14.6549.136","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.26.4.1333-1346.2006"},{"key":"2016081822022971000_44.14.6549.137","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajhg.2008.01.014"},{"key":"2016081822022971000_44.14.6549.138","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddr600"},{"key":"2016081822022971000_44.14.6549.139","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.3001777"},{"key":"2016081822022971000_44.14.6549.140","doi-asserted-by":"publisher","DOI":"10.1002\/emmm.201302567"},{"key":"2016081822022971000_44.14.6549.141","doi-asserted-by":"publisher","DOI":"10.1101\/gad.256644.114"},{"key":"2016081822022971000_44.14.6549.142","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002445"},{"key":"2016081822022971000_44.14.6549.143","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2012.0398"},{"key":"2016081822022971000_44.14.6549.144","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0812436106"},{"key":"2016081822022971000_44.14.6549.145","doi-asserted-by":"publisher","DOI":"10.1038\/nature10456"},{"key":"2016081822022971000_44.14.6549.146","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.3002847"},{"key":"2016081822022971000_44.14.6549.147","doi-asserted-by":"publisher","DOI":"10.1172\/JCI85908"},{"key":"2016081822022971000_44.14.6549.148","doi-asserted-by":"publisher","DOI":"10.1172\/JCI33355"},{"key":"2016081822022971000_44.14.6549.149","doi-asserted-by":"publisher","DOI":"10.1172\/JCI76175"},{"key":"2016081822022971000_44.14.6549.150","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2013.0449"},{"key":"2016081822022971000_44.14.6549.151","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkq731"},{"key":"2016081822022971000_44.14.6549.152","doi-asserted-by":"publisher","DOI":"10.1038\/bjc.2013.247"},{"key":"2016081822022971000_44.14.6549.153","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1172\/JCI82534","article-title":"Antisense oligonucleotide-mediated MDM4 exon 6 skipping impairs tumor growth","volume":"126","author":"Dewaele","year":"2016","journal-title":"J. Clin. Invest."},{"key":"2016081822022971000_44.14.6549.154","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1108482108"},{"key":"2016081822022971000_44.14.6549.155","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2012.58"},{"key":"2016081822022971000_44.14.6549.156","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2008.85"},{"key":"2016081822022971000_44.14.6549.157","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0033576"},{"key":"2016081822022971000_44.14.6549.158","doi-asserted-by":"publisher","DOI":"10.1096\/fj.12-213835"},{"key":"2016081822022971000_44.14.6549.159","doi-asserted-by":"crossref","first-page":"1060","DOI":"10.1002\/emmm.201202168","article-title":"Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice","volume":"5","author":"Gedicke-Hornung","year":"2013","journal-title":"EMBO Mol. Med."},{"key":"2016081822022971000_44.14.6549.160","doi-asserted-by":"publisher","DOI":"10.15252\/emmm.201505047"},{"key":"2016081822022971000_44.14.6549.161","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2015.144"},{"key":"2016081822022971000_44.14.6549.162","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2013.0457"},{"key":"2016081822022971000_44.14.6549.163","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2013.0452"},{"key":"2016081822022971000_44.14.6549.164","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2012.264"},{"key":"2016081822022971000_44.14.6549.165","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2010.212"},{"key":"2016081822022971000_44.14.6549.166","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2015.88"},{"key":"2016081822022971000_44.14.6549.167","first-page":"e206","article-title":"Antisense oligonucleotide-mediated suppression of Muscle Glycogen Synthase 1 synthesis as an approach for substrate reduction therapy of pompe disease","volume":"3","author":"Clayton","year":"2014","journal-title":"Mol. Ther."},{"key":"2016081822022971000_44.14.6549.168","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2013.04.019"}],"container-title":["Nucleic Acids Research"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/academic.oup.com\/nar\/article-pdf\/44\/14\/6549\/7628906\/gkw533.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2017,8,24]],"date-time":"2017-08-24T08:58:30Z","timestamp":1503565110000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/nar\/article-lookup\/doi\/10.1093\/nar\/gkw533"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,6,10]]},"references-count":168,"journal-issue":{"issue":"14","published-online":{"date-parts":[[2016,8,18]]},"published-print":{"date-parts":[[2016,8,19]]}},"alternative-id":["10.1093\/nar\/gkw533"],"URL":"https:\/\/doi.org\/10.1093\/nar\/gkw533","relation":{},"ISSN":["0305-1048","1362-4962"],"issn-type":[{"value":"0305-1048","type":"print"},{"value":"1362-4962","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,6,10]]}}}