{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,2]],"date-time":"2026-11-02T22:55:26Z","timestamp":1793660126277,"version":"4.5.1"},"reference-count":54,"publisher":"Oxford University Press (OUP)","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Hum. Mol. Genet."],"DOI":"10.1093\/hmg\/ddw118","type":"journal-article","created":{"date-parts":[[2016,4,23]],"date-time":"2016-04-23T02:59:04Z","timestamp":1461380344000},"page":"ddw118","source":"Crossref","is-referenced-by-count":31,"title":["<i>In vitro<\/i>and<i>in vivo<\/i>rescue of aberrant splicing in<i>CEP290<\/i>-associated LCA by antisense oligonucleotide delivery"],"prefix":"10.1093","author":[{"given":"Alejandro","family":"Garanto","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel C.","family":"Chung","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lonneke","family":"Duijkers","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julio C.","family":"Corral-Serrano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Muri\u00ebl","family":"Messchaert","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ru","family":"Xiao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean","family":"Bennett","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luk H.","family":"Vandenberghe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rob W.J.","family":"Collin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2016,4,22]]},"reference":[{"key":"2016051717335468000_ddw118v2.1","doi-asserted-by":"publisher","DOI":"10.1016\/j.survophthal.2004.04.003"},{"key":"2016051717335468000_ddw118v2.2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajo.2007.08.022"},{"key":"2016051717335468000_ddw118v2.3","doi-asserted-by":"publisher","DOI":"10.1016\/j.preteyeres.2008.05.003"},{"key":"2016051717335468000_ddw118v2.4","doi-asserted-by":"publisher","DOI":"10.1002\/humu.21337"},{"key":"2016051717335468000_ddw118v2.5","doi-asserted-by":"publisher","DOI":"10.1086\/507318"},{"key":"2016051717335468000_ddw118v2.6","doi-asserted-by":"publisher","DOI":"10.1093\/dnares\/4.2.141"},{"key":"2016051717335468000_ddw118v2.7","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.201006105"},{"key":"2016051717335468000_ddw118v2.8","doi-asserted-by":"publisher","DOI":"10.1002\/humu.21336"},{"key":"2016051717335468000_ddw118v2.9","doi-asserted-by":"publisher","DOI":"10.1002\/humu.9484"},{"key":"2016051717335468000_ddw118v2.10","first-page":"2160","article-title":"Frequency of CEP290 c.2991_1655A>G mutation in 175 Spanish families affected with Leber congenital amaurosis and early-onset retinitis pigmentosa","volume":"13","author":"Vallespin","year":"2007","journal-title":"Mol. Vis"},{"key":"2016051717335468000_ddw118v2.11","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2008.107"},{"key":"2016051717335468000_ddw118v2.12","doi-asserted-by":"publisher","DOI":"10.1001\/archophthalmol.2011.298"},{"key":"2016051717335468000_ddw118v2.13","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(09)61836-5"},{"key":"2016051717335468000_ddw118v2.14","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa0802315"},{"key":"2016051717335468000_ddw118v2.15","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa0802268"},{"key":"2016051717335468000_ddw118v2.16","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(13)62117-0"},{"key":"2016051717335468000_ddw118v2.17","doi-asserted-by":"publisher","DOI":"10.1016\/j.tig.2011.02.004"},{"key":"2016051717335468000_ddw118v2.18","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1011367"},{"key":"2016051717335468000_ddw118v2.19","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa073108"},{"key":"2016051717335468000_ddw118v2.20","doi-asserted-by":"publisher","DOI":"10.1007\/s10637-013-9935-x"},{"key":"2016051717335468000_ddw118v2.21","doi-asserted-by":"publisher","DOI":"10.1158\/1078-0432.CCR-03-0701"},{"key":"2016051717335468000_ddw118v2.22","doi-asserted-by":"publisher","DOI":"10.2174\/13816128113199990312"},{"key":"2016051717335468000_ddw118v2.23","doi-asserted-by":"publisher","DOI":"10.1177\/193229680900300440"},{"key":"2016051717335468000_ddw118v2.24","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2012.3"},{"key":"2016051717335468000_ddw118v2.25","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2012.21"},{"key":"2016051717335468000_ddw118v2.26","doi-asserted-by":"publisher","DOI":"10.4161\/cbt.1.4.4"},{"key":"2016051717335468000_ddw118v2.27","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2008.283"},{"key":"2016051717335468000_ddw118v2.28","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2005.08.008"},{"key":"2016051717335468000_ddw118v2.29","doi-asserted-by":"publisher","DOI":"10.1126\/science.1104297"},{"key":"2016051717335468000_ddw118v2.30","doi-asserted-by":"publisher","DOI":"10.1038\/ng.891"},{"key":"2016051717335468000_ddw118v2.31","doi-asserted-by":"publisher","DOI":"10.1016\/S0091-679X(08)94007-3"},{"key":"2016051717335468000_ddw118v2.32","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0079369"},{"key":"2016051717335468000_ddw118v2.33","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0053463"},{"key":"2016051717335468000_ddw118v2.34","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0054146"},{"key":"2016051717335468000_ddw118v2.35","doi-asserted-by":"publisher","DOI":"10.1038\/cddis.2010.40"},{"key":"2016051717335468000_ddw118v2.36","doi-asserted-by":"publisher","DOI":"10.1167\/iovs.13-13707"},{"key":"2016051717335468000_ddw118v2.37","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2012.280"},{"key":"2016051717335468000_ddw118v2.38","doi-asserted-by":"crossref","first-page":"120ra115.","DOI":"10.1126\/scitranslmed.3002865","article-title":"AAV2 gene therapy readministration in three adults with congenital blindness","volume":"4","author":"Bennett","year":"2012","journal-title":"Sci. Transl. Med"},{"key":"2016051717335468000_ddw118v2.39","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2014.8"},{"key":"2016051717335468000_ddw118v2.40","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2013.3"},{"key":"2016051717335468000_ddw118v2.41","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2014.39"},{"key":"2016051717335468000_ddw118v2.42","doi-asserted-by":"publisher","DOI":"10.1016\/j.visres.2007.07.027"},{"key":"2016051717335468000_ddw118v2.43","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddr022"},{"key":"2016051717335468000_ddw118v2.44","doi-asserted-by":"publisher","DOI":"10.1038\/nm.3106"},{"key":"2016051717335468000_ddw118v2.45","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2012.00296"},{"key":"2016051717335468000_ddw118v2.46","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2015.24"},{"key":"2016051717335468000_ddw118v2.47","doi-asserted-by":"publisher","DOI":"10.1167\/iovs.15-16400"},{"key":"2016051717335468000_ddw118v2.48","doi-asserted-by":"publisher","DOI":"10.1039\/C4MD00184B"},{"key":"2016051717335468000_ddw118v2.49","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.3005708"},{"key":"2016051717335468000_ddw118v2.50","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0062097"},{"key":"2016051717335468000_ddw118v2.51","doi-asserted-by":"publisher","DOI":"10.1002\/humu.20565"},{"key":"2016051717335468000_ddw118v2.52","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0092928"},{"key":"2016051717335468000_ddw118v2.53","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0100146"},{"key":"2016051717335468000_ddw118v2.54","unstructured":"Rasband W.S. (1997-2012) U. S. National Institutes of Health. Bethesda, MD. http:\/\/imagej.nih.gov\/ij\/, last accessed April 25, 2016."}],"container-title":["Human Molecular Genetics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/academic.oup.com\/hmg\/article\/25\/12\/2552\/2525743\/In-vitro-and-in-vivo-rescue-of-aberrant-splicing","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2017,6,24]],"date-time":"2017-06-24T11:36:53Z","timestamp":1498304213000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/hmg\/article-lookup\/doi\/10.1093\/hmg\/ddw118"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,4,22]]},"references-count":54,"alternative-id":["10.1093\/hmg\/ddw118"],"URL":"https:\/\/doi.org\/10.1093\/hmg\/ddw118","relation":{},"ISSN":["0964-6906","1460-2083"],"issn-type":[{"value":"0964-6906","type":"print"},{"value":"1460-2083","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,4,22]]}}}