{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T09:31:09Z","timestamp":1791192669655,"version":"4.1.0"},"reference-count":141,"publisher":"Oxford University Press (OUP)","issue":"4","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Brain"],"published-print":{"date-parts":[[2010,4,1]]},"DOI":"10.1093\/brain\/awq002","type":"journal-article","created":{"date-parts":[[2010,2,12]],"date-time":"2010-02-12T02:26:38Z","timestamp":1265941598000},"page":"957-972","source":"Crossref","is-referenced-by-count":86,"title":["RNA-targeted splice-correction therapy for neuromuscular disease"],"prefix":"10.1093","volume":"133","author":[{"given":"M. J. A.","family":"Wood","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. J.","family":"Gait","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H.","family":"Yin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2010,2,11]]},"reference":[{"issue":"10","key":"1_29193172","doi-asserted-by":"publisher","first-page":"1609","DOI":"10.1261\/rna.653607","volume":"13","year":"2007","journal-title":"RNA","ISSN":"https:\/\/id.crossref.org\/issn\/1355-8382","issn-type":"print"},{"issue":"2","key":"2_22524119","doi-asserted-by":"publisher","first-page":"135","DOI":"10.1002\/mus.20586","volume":"34","author":"Aartsma-Rus","year":"2006","journal-title":"Muscle &amp; nerve","ISSN":"https:\/\/id.crossref.org\/issn\/0148-639X","issn-type":"print"},{"issue":"3","key":"3_22493155","doi-asserted-by":"publisher","first-page":"401","DOI":"10.1016\/j.ymthe.2006.02.022","volume":"14","author":"Aartsma-Rus","year":"2006","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"key":"4_36582617","doi-asserted-by":"crossref","first-page":"284","DOI":"10.1089\/oli.2005.15.284","volume":"15","year":"2005","ISSN":"https:\/\/id.crossref.org\/issn\/1557-8526","issn-type":"print"},{"issue":"3","key":"5_33613249","doi-asserted-by":"publisher","first-page":"293","DOI":"10.1002\/humu.20918","volume":"30","author":"Aartsma-Rus","year":"2009","journal-title":"Human mutation","ISSN":"https:\/\/id.crossref.org\/issn\/1059-7794","issn-type":"print"},{"issue":"8","key":"6_17563117","doi-asserted-by":"publisher","first-page":"907","DOI":"10.1093\/hmg\/ddg100","volume":"12","author":"Aartsma-Rus","year":"2003","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"3","key":"7_32020845","doi-asserted-by":"publisher","first-page":"548","DOI":"10.1038\/mt.2008.205","volume":"17","author":"Aartsma-Rus","year":"2009","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"Pt 4","key":"8_29070520","doi-asserted-by":"publisher","first-page":"775","DOI":"10.1042\/BST0350775","volume":"35","author":"Abes","year":"2007","journal-title":"Biochemical Society Transactions","ISSN":"https:\/\/id.crossref.org\/issn\/1470-8752","issn-type":"print"},{"issue":"4","key":"9_14634686","doi-asserted-by":"publisher","first-page":"283","DOI":"10.1038\/ng0493-283","volume":"3","author":"Ahn","year":"1993","journal-title":"Nature genetics","ISSN":"https:\/\/id.crossref.org\/issn\/1061-4036","issn-type":"print"},{"issue":"2","key":"10_21594959","doi-asserted-by":"publisher","first-page":"175","DOI":"10.1038\/nm1345","volume":"12","author":"Alter","year":"2006","journal-title":"Nature medicine","ISSN":"https:\/\/id.crossref.org\/issn\/1078-8956","issn-type":"print"},{"issue":"2-3","key":"11_18586792","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1016\/j.bbadis.2004.08.010","volume":"1739","author":"Andreadis","year":"2005","journal-title":"Biochimica et Biophysica Acta. Protein Structure and Molecular Enzymology","ISSN":"https:\/\/id.crossref.org\/issn\/0006-3002","issn-type":"print"},{"issue":"9","key":"12_29566377","doi-asserted-by":"publisher","first-page":"798","DOI":"10.1089\/hum.2006.061","volume":"18","author":"Arechavala-Gomeza","year":"2007","journal-title":"Human gene therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1043-0342","issn-type":"print"},{"issue":"2","key":"13_6411442","doi-asserted-by":"publisher","first-page":"237","DOI":"10.1093\/hmg\/9.2.237","volume":"9","author":"Ars","year":"2000","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"9","key":"14_34092700","doi-asserted-by":"publisher","first-page":"1600","DOI":"10.1093\/hmg\/ddp076","volume":"18","year":"2009","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"1","key":"15_22387466","doi-asserted-by":"publisher","first-page":"37","DOI":"10.1016\/j.cell.2006.06.023","volume":"126","author":"Blencowe","year":"2006","journal-title":"Cell","ISSN":"https:\/\/id.crossref.org\/issn\/0092-8674","issn-type":"print"},{"issue":"2","key":"16_18404161","doi-asserted-by":"publisher","first-page":"232","DOI":"10.1016\/j.ymthe.2004.05.031","volume":"10","author":"Bremmer-Bout","year":"2004","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"4","key":"17_15683181","doi-asserted-by":"publisher","first-page":"359","DOI":"10.1016\/0960-8966(94)90072-8","volume":"4","author":"Bresolin","year":"1994","journal-title":"Neuromuscular disorders : NMD","ISSN":"https:\/\/id.crossref.org\/issn\/0960-8966","issn-type":"print"},{"issue":"4","key":"18_13172227","doi-asserted-by":"publisher","first-page":"1189","DOI":"10.1073\/pnas.81.4.1189","volume":"81","author":"Bulfield","year":"1984","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"issue":"13","key":"19_28895919","doi-asserted-by":"publisher","first-page":"4369","DOI":"10.1093\/nar\/gkm447","volume":"35","year":"2007","journal-title":"Nucleic Acids Research","ISSN":"https:\/\/id.crossref.org\/issn\/0305-1048","issn-type":"print"},{"issue":"8","key":"20_35254749","doi-asserted-by":"publisher","first-page":"597","DOI":"10.1038\/nrn2670","volume":"10","author":"Burghes","year":"2009","journal-title":"Nature reviews. Neuroscience","ISSN":"https:\/\/id.crossref.org\/issn\/1471-003X","issn-type":"print"},{"key":"21_36582618","first-page":"166","volume":"13","year":"2003","journal-title":"THTH JUNE NAARDEN THE NETHERLANDS NEUROMUSCUL DISORD"},{"issue":"8-9","key":"22_18305105","doi-asserted-by":"publisher","first-page":"526","DOI":"10.1016\/j.nmd.2004.05.006","volume":"14","author":"Bushby","year":"2004","journal-title":"Neuromuscular disorders : NMD","ISSN":"https:\/\/id.crossref.org\/issn\/0960-8966","issn-type":"print"},{"issue":"2","key":"23_17483261","doi-asserted-by":"publisher","first-page":"120","DOI":"10.1038\/nsb887","volume":"10","author":"Cartegni","year":"2003","journal-title":"Nature structural biology","ISSN":"https:\/\/id.crossref.org\/issn\/1072-8368","issn-type":"print"},{"issue":"1","key":"24_21235049","doi-asserted-by":"publisher","first-page":"63","DOI":"10.1086\/498853","volume":"78","author":"Agnew","year":"2006","journal-title":"American journal of human genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0002-9297","issn-type":"print"},{"issue":"13","key":"25_14537566","first-page":"3646","volume":"34","author":"Cibis","year":"1993","journal-title":"Investigative Ophthalmology &amp; Visual Science","ISSN":"https:\/\/id.crossref.org\/issn\/0020-9988","issn-type":"print"},{"issue":"8","key":"26_29116272","doi-asserted-by":"publisher","first-page":"1471","DOI":"10.1038\/sj.mt.6300222","volume":"15","author":"Coady","year":"2007","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"6","key":"27_28673083","doi-asserted-by":"publisher","first-page":"1064","DOI":"10.1086\/518127","volume":"80","author":"Costa","year":"2007","journal-title":"American journal of human genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0002-9297","issn-type":"print"},{"issue":"3","key":"28_16864571","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1097\/00001573-199705000-00015","volume":"12","author":"Cox","year":"1997","journal-title":"Current opinion in cardiology","ISSN":"https:\/\/id.crossref.org\/issn\/0268-4705","issn-type":"print"},{"issue":"4390","key":"29_4778119","doi-asserted-by":"publisher","first-page":"264","DOI":"10.1126\/science.373120","volume":"204","author":"Crick","year":"1979","journal-title":"Science","ISSN":"https:\/\/id.crossref.org\/issn\/0036-8075","issn-type":"print"},{"issue":"10","key":"30_10815411","doi-asserted-by":"publisher","first-page":"5598","DOI":"10.1073\/pnas.96.10.5598","volume":"96","author":"D'Souza","year":"1999","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"issue":"8","key":"31_35476744","doi-asserted-by":"publisher","first-page":"e1000600","DOI":"10.1371\/journal.pgen.1000600","volume":"5","author":"Daughters","year":"2009","ISSN":"https:\/\/id.crossref.org\/issn\/1553-7390","issn-type":"print"},{"issue":"10","key":"32_22631796","doi-asserted-by":"publisher","first-page":"762","DOI":"10.1038\/nrm2024","volume":"7","author":"Davies","year":"2006","journal-title":"Nature reviews. Molecular cell biology","ISSN":"https:\/\/id.crossref.org\/issn\/1471-0072","issn-type":"print"},{"issue":"14","key":"33_19673199","doi-asserted-by":"publisher","first-page":"9456","DOI":"10.1073\/pnas.142302299","volume":"99","year":"2002","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"issue":"10","key":"34_21660510","doi-asserted-by":"publisher","first-page":"3758","DOI":"10.1073\/pnas.0508917103","volume":"103","year":"2006","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"issue":"14","key":"35_14704228","doi-asserted-by":"crossref","first-page":"10444","DOI":"10.1016\/S0021-9258(17)34080-2","volume":"269","author":"Derossi","year":"1994","journal-title":"Journal of Biological Chemistry","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9258","issn-type":"print"},{"issue":"11","key":"36_35044613","doi-asserted-by":"publisher","first-page":"1307","DOI":"10.1089\/hum.2008.067","volume":"19","author":"Dickson","year":"2008","journal-title":"Human gene therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1043-0342","issn-type":"print"},{"issue":"18","key":"37_14453547","doi-asserted-by":"publisher","first-page":"8673","DOI":"10.1073\/pnas.90.18.8673","volume":"90","author":"Dominski","year":"1993","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"key":"38_36582619","first-page":"R253","volume":"152","year":"2006","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"7","key":"39_6033405","doi-asserted-by":"publisher","first-page":"1083","DOI":"10.1093\/hmg\/7.7.1083","volume":"7","author":"Dunckley","year":"1998","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"key":"40_22174631","doi-asserted-by":"publisher","first-page":"3","DOI":"10.1186\/1479-0556-4-3","volume":"4","author":"Fall","year":"2006","ISSN":"https:\/\/id.crossref.org\/issn\/1479-0556","issn-type":"print"},{"issue":"5","key":"41_33870443","doi-asserted-by":"publisher","first-page":"659","DOI":"10.1007\/s00246-008-9359-0","volume":"30","author":"Finsterer","year":"2009","journal-title":"Pediatric cardiology","ISSN":"https:\/\/id.crossref.org\/issn\/0172-0643","issn-type":"print"},{"issue":"1","key":"42_17503185","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1159\/000068446","volume":"99","author":"Finsterer","year":"2003","journal-title":"Cardiology","ISSN":"https:\/\/id.crossref.org\/issn\/0008-6312","issn-type":"print"},{"issue":"6","key":"43_15507845","doi-asserted-by":"crossref","first-page":"2425","DOI":"10.1172\/JCI117250","volume":"93","author":"Fitzgerald","year":"1994","journal-title":"Journal of Clinical Investigation","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9738","issn-type":"print"},{"issue":"5","key":"44_13985735","doi-asserted-by":"crossref","first-page":"589","DOI":"10.1111\/j.1469-8749.1986.tb03900.x","volume":"28","author":"Fitzpatrick","year":"1986","journal-title":"Developmental medicine and child neurology","ISSN":"https:\/\/id.crossref.org\/issn\/0012-1622","issn-type":"print"},{"issue":"2","key":"45_21594568","doi-asserted-by":"publisher","first-page":"207","DOI":"10.1002\/jgm.838","volume":"8","author":"Fletcher","year":"2006","journal-title":"The journal of gene medicine","ISSN":"https:\/\/id.crossref.org\/issn\/1099-498X","issn-type":"print"},{"issue":"9","key":"46_29272000","doi-asserted-by":"publisher","first-page":"1587","DOI":"10.1038\/sj.mt.6300245","volume":"15","author":"Fletcher","year":"2007","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"1","key":"47_33488572","doi-asserted-by":"publisher","first-page":"59","DOI":"10.1038\/nbt.1515","volume":"27","author":"Foust","year":"2009","journal-title":"Nature biotechnology","ISSN":"https:\/\/id.crossref.org\/issn\/1087-0156","issn-type":"print"},{"issue":"15","key":"48_17731078","doi-asserted-by":"publisher","first-page":"1801","DOI":"10.1093\/hmg\/ddg196","volume":"12","author":"Gebski","year":"2003","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"5702","key":"49_18551752","doi-asserted-by":"publisher","first-page":"1796","DOI":"10.1126\/science.1104297","volume":"306","year":"2004","journal-title":"Science","ISSN":"https:\/\/id.crossref.org\/issn\/0036-8075","issn-type":"print"},{"issue":"21","key":"50_10822555","doi-asserted-by":"publisher","first-page":"15134","DOI":"10.1074\/jbc.274.21.15134","volume":"274","author":"Grover","year":"1999","journal-title":"Journal of Biological Chemistry","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9258","issn-type":"print"},{"issue":"3","key":"51_33512307","doi-asserted-by":"publisher","first-page":"257","DOI":"10.1002\/jgm.1288","volume":"11","author":"Heemskerk","year":"2009","journal-title":"The journal of gene medicine","ISSN":"https:\/\/id.crossref.org\/issn\/1099-498X","issn-type":"print"},{"issue":"3","key":"52_29889348","doi-asserted-by":"publisher","first-page":"1211","DOI":"10.1074\/jbc.R700035200","volume":"283","year":"2008","journal-title":"Journal of Biological Chemistry","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9258","issn-type":"print"},{"issue":"3","key":"53_22038127","first-page":"209","volume":"24","author":"Hirst","year":"2005","journal-title":"Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology \/ edited by the Gaetano Conte Academy for the study of striated muscle diseases","ISSN":"https:\/\/id.crossref.org\/issn\/1128-2460","issn-type":"print"},{"issue":"26","key":"54_30104688","doi-asserted-by":"publisher","first-page":"2719","DOI":"10.1056\/NEJMe0707795","volume":"357","author":"Hoffman","year":"2007","journal-title":"New England Journal of Medicine","ISSN":"https:\/\/id.crossref.org\/issn\/0028-4793","issn-type":"print"},{"issue":"6150","key":"55_14344539","doi-asserted-by":"publisher","first-page":"754","DOI":"10.1038\/330754a0","volume":"330","author":"Hoffman","year":"1987","journal-title":"Nature; Physical Science (London)","ISSN":"https:\/\/id.crossref.org\/issn\/1476-4687","issn-type":"print"},{"issue":"4","key":"56_27939030","doi-asserted-by":"publisher","first-page":"e73","DOI":"10.1371\/journal.pbio.0050073","volume":"5","author":"Hua","year":"2007","journal-title":"PLoS biology","ISSN":"https:\/\/id.crossref.org\/issn\/1544-9173","issn-type":"print"},{"issue":"4","key":"57_30803184","doi-asserted-by":"publisher","first-page":"834","DOI":"10.1016\/j.ajhg.2008.01.014","volume":"82","author":"Hua","year":"2008","journal-title":"American journal of human genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0002-9297","issn-type":"print"},{"issue":"20","key":"58_32095400","doi-asserted-by":"publisher","first-page":"6418","DOI":"10.1093\/nar\/gkn671","volume":"36","year":"2008","journal-title":"Nucleic Acids Research","ISSN":"https:\/\/id.crossref.org\/issn\/0305-1048","issn-type":"print"},{"issue":"1","key":"59_31820110","doi-asserted-by":"publisher","first-page":"31","DOI":"10.1093\/nass\/nrn016","volume":"52","year":"2008","journal-title":"Nucleic Acids Symposium Series","ISSN":"https:\/\/id.crossref.org\/issn\/0261-3166","issn-type":"print"},{"issue":"9","key":"60_31783724","doi-asserted-by":"publisher","first-page":"1624","DOI":"10.1038\/mt.2008.120","volume":"16","author":"Jearawiriyapaisarn","year":"2008","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"key":"61_34225799","first-page":"739","volume":"39","year":"2003","ISSN":"https:\/\/id.crossref.org\/issn\/1097-4199","issn-type":"print"},{"issue":"22","key":"62_10342389","doi-asserted-by":"crossref","first-page":"2586","DOI":"10.1161\/01.CIR.101.22.2586","volume":"101","author":"Kaprielian","year":"2000","journal-title":"Circulation","ISSN":"https:\/\/id.crossref.org\/issn\/0009-7322","issn-type":"print"},{"issue":"4","key":"63_17770572","doi-asserted-by":"publisher","first-page":"460","DOI":"10.1038\/ng1207","volume":"34","author":"Kashima","year":"2003","journal-title":"Nature genetics","ISSN":"https:\/\/id.crossref.org\/issn\/1061-4036","issn-type":"print"},{"issue":"10","key":"64_35505021","doi-asserted-by":"publisher","first-page":"918","DOI":"10.1016\/S1474-4422(09)70211-X","volume":"8","author":"Kinali","year":"2009","journal-title":"Lancet. Neurology","ISSN":"https:\/\/id.crossref.org\/issn\/1474-4422","issn-type":"print"},{"issue":"5758","key":"65_21148000","doi-asserted-by":"publisher","first-page":"230","DOI":"10.1126\/science.1118265","volume":"311","year":"2006","journal-title":"Science","ISSN":"https:\/\/id.crossref.org\/issn\/0036-8075","issn-type":"print"},{"issue":"2","key":"66_10095371","doi-asserted-by":"crossref","first-page":"349","DOI":"10.1161\/01.RES.72.2.349","volume":"72","author":"Klietsch","year":"1993","journal-title":"Circulation Research","ISSN":"https:\/\/id.crossref.org\/issn\/0009-7330","issn-type":"print"},{"issue":"2","key":"67_13270745","doi-asserted-by":"publisher","first-page":"191","DOI":"10.1007\/BF02409661","volume":"14","author":"Komoto","year":"1984","journal-title":"Journal of autism and developmental disorders","ISSN":"https:\/\/id.crossref.org\/issn\/0162-3257","issn-type":"print"},{"issue":"1","key":"68_29675686","doi-asserted-by":"publisher","first-page":"68","DOI":"10.1016\/j.molcel.2007.07.027","volume":"28","author":"Kuyumcu-Martinez","year":"2007","journal-title":"Molecular cell","ISSN":"https:\/\/id.crossref.org\/issn\/1097-2765","issn-type":"print"},{"issue":"6","key":"69_34414189","doi-asserted-by":"publisher","first-page":"1001","DOI":"10.1016\/j.cell.2009.03.006","volume":"136","author":"Lagier-Tourenne","year":"2009","journal-title":"Cell","ISSN":"https:\/\/id.crossref.org\/issn\/0092-8674","issn-type":"print"},{"issue":"3","key":"70_16832250","doi-asserted-by":"publisher","first-page":"265","DOI":"10.1038\/ng0797-265","volume":"16","author":"Lefebvre","year":"1997","journal-title":"Nature genetics","ISSN":"https:\/\/id.crossref.org\/issn\/1061-4036","issn-type":"print"},{"issue":"7221","key":"71_32505960","doi-asserted-by":"publisher","first-page":"464","DOI":"10.1038\/nature07488","volume":"456","author":"Licatalosi","year":"2008","journal-title":"Nature; Physical Science (London)","ISSN":"https:\/\/id.crossref.org\/issn\/1476-4687","issn-type":"print"},{"issue":"6303","key":"72_9254484","doi-asserted-by":"publisher","first-page":"725","DOI":"10.1038\/348725a0","volume":"348","author":"Lidov","year":"1990","journal-title":"Nature; Physical Science (London)","ISSN":"https:\/\/id.crossref.org\/issn\/1476-4687","issn-type":"print"},{"issue":"48","key":"73_19551658","doi-asserted-by":"publisher","first-page":"45476","DOI":"10.1074\/jbc.M107632200","volume":"276","year":"2001","journal-title":"Journal of Biological Chemistry","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9258","issn-type":"print"},{"issue":"13","key":"74_22157499","doi-asserted-by":"publisher","first-page":"2087","DOI":"10.1093\/hmg\/ddl132","volume":"15","year":"2006","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"8","key":"75_17748711","doi-asserted-by":"publisher","first-page":"1009","DOI":"10.1038\/nm897","volume":"9","author":"Lu","year":"2003","journal-title":"Nature medicine","ISSN":"https:\/\/id.crossref.org\/issn\/1078-8956","issn-type":"print"},{"issue":"5","key":"76_35890872","doi-asserted-by":"publisher","first-page":"985","DOI":"10.1083\/jcb.148.5.985","volume":"148","year":"2000","journal-title":"The Journal of Cell Biology","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9525","issn-type":"print"},{"issue":"1","key":"77_19688750","doi-asserted-by":"publisher","first-page":"198","DOI":"10.1073\/pnas.0406700102","volume":"102","year":"2005","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"issue":"6","key":"78_21485826","doi-asserted-by":"publisher","first-page":"1013","DOI":"10.1016\/j.ymthe.2005.08.022","volume":"12","author":"Madocsai","year":"2005","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"9","key":"79_22559230","doi-asserted-by":"publisher","first-page":"1066","DOI":"10.1038\/ng1857","volume":"38","author":"Mahadevan","year":"2006","journal-title":"Nature genetics","ISSN":"https:\/\/id.crossref.org\/issn\/1061-4036","issn-type":"print"},{"issue":"1","key":"80_19670948","doi-asserted-by":"publisher","first-page":"42","DOI":"10.1073\/pnas.011408598","volume":"98","year":"2001","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"issue":"11","key":"81_21946063","doi-asserted-by":"publisher","first-page":"1808","DOI":"10.1093\/hmg\/ddl103","volume":"15","year":"2006","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"5","key":"82_31424463","doi-asserted-by":"publisher","first-page":"365","DOI":"10.1016\/j.nmd.2008.03.002","volume":"18","author":"Markham","year":"2008","journal-title":"Neuromuscular disorders : NMD","ISSN":"https:\/\/id.crossref.org\/issn\/0960-8966","issn-type":"print"},{"issue":"8","key":"83_29115998","doi-asserted-by":"publisher","first-page":"1479","DOI":"10.1038\/sj.mt.6300200","volume":"15","author":"Marquis","year":"2007","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"5","key":"84_18922394","doi-asserted-by":"publisher","first-page":"386","DOI":"10.1038\/nrm1645","volume":"6","author":"Matlin","year":"2005","journal-title":"Nature reviews. Molecular cell biology","ISSN":"https:\/\/id.crossref.org\/issn\/1471-0072","issn-type":"print"},{"issue":"19","key":"85_22620716","doi-asserted-by":"publisher","first-page":"1373","DOI":"10.1038\/sj.gt.3302800","volume":"13","author":"McClorey","year":"2006","journal-title":"Gene therapy","ISSN":"https:\/\/id.crossref.org\/issn\/0969-7128","issn-type":"print"},{"issue":"3","key":"87_32626599","doi-asserted-by":"publisher","first-page":"546","DOI":"10.1093\/hmg\/ddn382","volume":"18","year":"2009","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"1","key":"88_11453630","doi-asserted-by":"publisher","first-page":"13","DOI":"10.1038\/ng0102-13","volume":"30","author":"Modrek","year":"2002","journal-title":"Nature genetics","ISSN":"https:\/\/id.crossref.org\/issn\/1061-4036","issn-type":"print"},{"issue":"7","key":"89_22341777","doi-asserted-by":"publisher","first-page":"758","DOI":"10.1038\/ng1827","volume":"38","author":"Moseley","year":"2006","journal-title":"Nature genetics","ISSN":"https:\/\/id.crossref.org\/issn\/1061-4036","issn-type":"print"},{"issue":"33","key":"90_35379840","doi-asserted-by":"publisher","first-page":"13915","DOI":"10.1073\/pnas.0905780106","volume":"106","year":"2009","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"issue":"12","key":"91_17924210","doi-asserted-by":"publisher","first-page":"731","DOI":"10.1016\/S1474-4422(03)00585-4","volume":"2","author":"Muntoni","year":"2003","journal-title":"Lancet. Neurology","ISSN":"https:\/\/id.crossref.org\/issn\/1474-4422","issn-type":"print"},{"issue":"4","key":"92_19270484","doi-asserted-by":"publisher","first-page":"959","DOI":"10.1021\/bc0501045","volume":"16","author":"Nelson","year":"2005","journal-title":"Bioconjugate chemistry","ISSN":"https:\/\/id.crossref.org\/issn\/1043-1802","issn-type":"print"},{"issue":"12","key":"93_32406817","doi-asserted-by":"publisher","first-page":"7419","DOI":"10.1074\/jbc.R800025200","volume":"284","year":"2009","journal-title":"Journal of Biological Chemistry","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9258","issn-type":"print"},{"issue":"1","key":"94_33699954","doi-asserted-by":"publisher","first-page":"72","DOI":"10.1016\/j.jjcc.2008.08.013","volume":"53","author":"Ogata","year":"2009","journal-title":"Journal of cardiology","ISSN":"https:\/\/id.crossref.org\/issn\/0914-5087","issn-type":"print"},{"key":"95_36383046","first-page":"R162","volume":"152","year":"2006","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"4","key":"96_16985339","doi-asserted-by":"publisher","first-page":"426","DOI":"10.1038\/ng858","volume":"30","author":"Pagani","year":"2002","journal-title":"Nature genetics","ISSN":"https:\/\/id.crossref.org\/issn\/1061-4036","issn-type":"print"},{"issue":"5364","key":"97_5984345","doi-asserted-by":"publisher","first-page":"737","DOI":"10.1126\/science.280.5364.737","volume":"280","author":"Philips","year":"1998","journal-title":"Science","ISSN":"https:\/\/id.crossref.org\/issn\/0036-8075","issn-type":"print"},{"issue":"17","key":"98_16208032","doi-asserted-by":"publisher","first-page":"1335","DOI":"10.1001\/jama.1996.03530410049032","volume":"275","author":"Politano","year":"1996","journal-title":"JAMA","ISSN":"https:\/\/id.crossref.org\/issn\/0098-7484","issn-type":"print"},{"issue":"5","key":"99_9820523","doi-asserted-by":"publisher","first-page":"997","DOI":"10.1083\/jcb.117.5.997","volume":"117","author":"Porter","year":"1992","journal-title":"The Journal of Cell Biology","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9525","issn-type":"print"},{"issue":"2","key":"100_16397583","doi-asserted-by":"publisher","first-page":"445","DOI":"10.1006\/bbrc.1996.1375","volume":"226","author":"Pramono","year":"1996","journal-title":"Biochemical and biophysical research communications","ISSN":"https:\/\/id.crossref.org\/issn\/0006-291X","issn-type":"print"},{"key":"101_22286990","doi-asserted-by":"publisher","first-page":"259","DOI":"10.1146\/annurev.neuro.29.051605.113014","volume":"29","author":"Ranum","year":"2006","journal-title":"Annual review of neuroscience","ISSN":"https:\/\/id.crossref.org\/issn\/0147-006X","issn-type":"print"},{"issue":"5","key":"102_34150380","doi-asserted-by":"publisher","first-page":"820","DOI":"10.1038\/mt.2009.8","volume":"17","author":"Rimessi","year":"2009","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"5","key":"103_35885376","doi-asserted-by":"publisher","first-page":"1209","DOI":"10.1083\/jcb.150.5.1209","volume":"150","year":"2000","journal-title":"The Journal of Cell Biology","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9525","issn-type":"print"},{"issue":"3","key":"104_16392705","doi-asserted-by":"publisher","first-page":"642","DOI":"10.1016\/S0002-8703(96)90250-1","volume":"132","author":"Saito","year":"1996","journal-title":"American heart journal","ISSN":"https:\/\/id.crossref.org\/issn\/0002-8703","issn-type":"print"},{"key":"105_36582620","first-page":"219","volume":"44","year":"2004","ISSN":"https:\/\/id.crossref.org\/issn\/1097-4199","issn-type":"print"},{"issue":"1","key":"106_35242706","doi-asserted-by":"publisher","first-page":"25","DOI":"10.1016\/j.ajhg.2009.06.003","volume":"85","author":"Schultz","year":"2009","journal-title":"American journal of human genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0002-9297","issn-type":"print"},{"issue":"1","key":"107_32212793","doi-asserted-by":"publisher","first-page":"124","DOI":"10.1093\/brain\/awn253","volume":"132","year":"2009","journal-title":"Brain","ISSN":"https:\/\/id.crossref.org\/issn\/0006-8950","issn-type":"print"},{"issue":"4","key":"108_34173694","doi-asserted-by":"publisher","first-page":"577","DOI":"10.1016\/j.cell.2009.02.007","volume":"136","author":"Sharp","year":"2009","journal-title":"Cell","ISSN":"https:\/\/id.crossref.org\/issn\/0092-8674","issn-type":"print"},{"issue":"4912","key":"109_5302120","doi-asserted-by":"publisher","first-page":"1578","DOI":"10.1126\/science.2662404","volume":"244","author":"Sicinski","year":"1989","journal-title":"Science","ISSN":"https:\/\/id.crossref.org\/issn\/0036-8075","issn-type":"print"},{"issue":"3","key":"110_34830571","doi-asserted-by":"crossref","first-page":"341","DOI":"10.4161\/rna.6.3.8723","volume":"6","author":"Singh","year":"2009","ISSN":"https:\/\/id.crossref.org\/issn\/1555-8584","issn-type":"print"},{"key":"111_34553042","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/1477-3155-7-1","volume":"7","author":"Sirsi","year":"2009","ISSN":"https:\/\/id.crossref.org\/issn\/1477-3155","issn-type":"print"},{"issue":"7","key":"112_19684904","doi-asserted-by":"publisher","first-page":"4114","DOI":"10.1073\/pnas.0633863100","volume":"100","year":"2003","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"issue":"3","key":"113_31332646","doi-asserted-by":"publisher","first-page":"555","DOI":"10.1093\/jac\/dkn221","volume":"62","year":"2008","journal-title":"Journal of Antimicrobial Chemotherapy","ISSN":"https:\/\/id.crossref.org\/issn\/0305-7453","issn-type":"print"},{"issue":"2","key":"114_15772471","doi-asserted-by":"crossref","first-page":"515","DOI":"10.1172\/JCI117693","volume":"95","author":"Takeshima","year":"1995","journal-title":"Journal of Clinical Investigation","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9738","issn-type":"print"},{"issue":"2","key":"115_11225361","doi-asserted-by":"publisher","first-page":"189","DOI":"10.1055\/s-2001-15264","volume":"21","author":"Talbot","year":"2001","journal-title":"Seminars in neurology","ISSN":"https:\/\/id.crossref.org\/issn\/0271-8235","issn-type":"print"},{"issue":"4","key":"116_31143089","doi-asserted-by":"publisher","first-page":"572","DOI":"10.1016\/j.cell.2008.05.002","volume":"133","author":"Talbot","year":"2008","journal-title":"Cell","ISSN":"https:\/\/id.crossref.org\/issn\/0092-8674","issn-type":"print"},{"issue":"2","key":"117_15842127","doi-asserted-by":"publisher","first-page":"184","DOI":"10.1038\/ng0295-184","volume":"9","author":"Tennyson","year":"1995","journal-title":"Nature genetics","ISSN":"https:\/\/id.crossref.org\/issn\/1061-4036","issn-type":"print"},{"issue":"6","key":"118_10823994","doi-asserted-by":"publisher","first-page":"1617","DOI":"10.1086\/302418","volume":"64","author":"Teraoka","year":"1999","journal-title":"American journal of human genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0002-9297","issn-type":"print"},{"issue":"6607","key":"119_16526245","doi-asserted-by":"publisher","first-page":"349","DOI":"10.1038\/384349a0","volume":"384","author":"Tinsley","year":"1996","journal-title":"Nature; Physical Science (London)","ISSN":"https:\/\/id.crossref.org\/issn\/1476-4687","issn-type":"print"},{"issue":"5","key":"120_30959359","doi-asserted-by":"publisher","first-page":"832","DOI":"10.1038\/mt.2008.52","volume":"16","author":"Townsend","year":"2008","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"10","key":"121_17841720","doi-asserted-by":"publisher","first-page":"774","DOI":"10.1038\/nrg1180","volume":"4","author":"van Deutekom","year":"2003","journal-title":"Nature reviews. Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/1471-0056","issn-type":"print"},{"issue":"15","key":"122_11248645","doi-asserted-by":"publisher","first-page":"1547","DOI":"10.1093\/hmg\/10.15.1547","volume":"10","author":"van Deutekom","year":"2001","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"26","key":"123_30104682","doi-asserted-by":"publisher","first-page":"2677","DOI":"10.1056\/NEJMoa073108","volume":"357","author":"van Deutekom","year":"2007","journal-title":"New England Journal of Medicine","ISSN":"https:\/\/id.crossref.org\/issn\/0028-4793","issn-type":"print"},{"key":"124_32760659","doi-asserted-by":"publisher","first-page":"105","DOI":"10.1186\/1471-2350-9-105","volume":"9","author":"van Vliet","year":"2008","journal-title":"BMC medical genetics [electronic resource]","ISSN":"https:\/\/id.crossref.org\/issn\/1471-2350","issn-type":"print"},{"issue":"25","key":"125_16814943","doi-asserted-by":"publisher","first-page":"16010","DOI":"10.1074\/jbc.272.25.16010","volume":"272","author":"Vives","year":"1997","journal-title":"Journal of Biological Chemistry","ISSN":"https:\/\/id.crossref.org\/issn\/0021-9258","issn-type":"print"},{"issue":"44","key":"126_35733656","doi-asserted-by":"publisher","first-page":"18551","DOI":"10.1073\/pnas.0903234106","volume":"106","year":"2009","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"issue":"7140","key":"127_28522255","doi-asserted-by":"publisher","first-page":"87","DOI":"10.1038\/nature05756","volume":"447","author":"Welch","year":"2007","journal-title":"Nature; Physical Science (London)","ISSN":"https:\/\/id.crossref.org\/issn\/1476-4687","issn-type":"print"},{"issue":"5938","key":"128_35285514","doi-asserted-by":"publisher","first-page":"336","DOI":"10.1126\/science.1173110","volume":"325","author":"Wheeler","year":"2009","journal-title":"Science","ISSN":"https:\/\/id.crossref.org\/issn\/0036-8075","issn-type":"print"},{"issue":"3","key":"129_18066089","doi-asserted-by":"crossref","first-page":"154","DOI":"10.1111\/j.1469-8749.2004.tb00466.x","volume":"46","author":"Wicksell","year":"2004","journal-title":"Developmental medicine and child neurology","ISSN":"https:\/\/id.crossref.org\/issn\/0012-1622","issn-type":"print"},{"issue":"24","key":"130_35044582","doi-asserted-by":"publisher","first-page":"7633","DOI":"10.1523\/JNEUROSCI.0950-09.2009","volume":"29","author":"Williams","year":"2009","journal-title":"Journal of Neuroscience","ISSN":"https:\/\/id.crossref.org\/issn\/0270-6474","issn-type":"print"},{"issue":"7","key":"131_28921047","doi-asserted-by":"publisher","first-page":"1288","DOI":"10.1038\/sj.mt.6300095","volume":"15","author":"Wilton","year":"2007","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"5","key":"132_10889204","doi-asserted-by":"publisher","first-page":"330","DOI":"10.1016\/S0960-8966(99)00010-3","volume":"9","author":"Wilton","year":"1999","journal-title":"Neuromuscular disorders : NMD","ISSN":"https:\/\/id.crossref.org\/issn\/0960-8966","issn-type":"print"},{"issue":"5","key":"133_34310245","doi-asserted-by":"publisher","first-page":"864","DOI":"10.1038\/mt.2009.38","volume":"17","author":"Wu","year":"2009","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"39","key":"134_31963421","doi-asserted-by":"publisher","first-page":"14814","DOI":"10.1073\/pnas.0805676105","volume":"105","year":"2008","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"},{"issue":"7053","key":"135_19335485","doi-asserted-by":"publisher","first-page":"1025","DOI":"10.1038\/nature03844","volume":"436","author":"Yasuda","year":"2005","journal-title":"Nature; Physical Science (London)","ISSN":"https:\/\/id.crossref.org\/issn\/1476-4687","issn-type":"print"},{"issue":"1","key":"136_30126440","doi-asserted-by":"publisher","first-page":"38","DOI":"10.1038\/sj.mt.6300329","volume":"16","author":"Yin","year":"2008","journal-title":"Molecular therapy : the journal of the American Society of Gene Therapy","ISSN":"https:\/\/id.crossref.org\/issn\/1525-0016","issn-type":"print"},{"issue":"22","key":"137_35420741","doi-asserted-by":"publisher","first-page":"4405","DOI":"10.1093\/hmg\/ddp395","volume":"18","year":"2009","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"24","key":"138_31882002","doi-asserted-by":"publisher","first-page":"3909","DOI":"10.1093\/hmg\/ddn293","volume":"17","year":"2008","journal-title":"Human Molecular Genetics","ISSN":"https:\/\/id.crossref.org\/issn\/0964-6906","issn-type":"print"},{"issue":"6","key":"139_34319657","doi-asserted-by":"publisher","first-page":"667","DOI":"10.1002\/ana.21627","volume":"65","author":"Yokota","year":"2009","journal-title":"Annals of neurology","ISSN":"https:\/\/id.crossref.org\/issn\/0364-5134","issn-type":"print"},{"issue":"20","key":"140_11396121","doi-asserted-by":"publisher","first-page":"1532","DOI":"10.1038\/sj.gt.3301550","volume":"8","author":"Zhang","year":"2001","journal-title":"Gene therapy","ISSN":"https:\/\/id.crossref.org\/issn\/0969-7128","issn-type":"print"},{"issue":"4","key":"141_31143093","doi-asserted-by":"publisher","first-page":"585","DOI":"10.1016\/j.cell.2008.03.031","volume":"133","author":"Zhang","year":"2008","journal-title":"Cell","ISSN":"https:\/\/id.crossref.org\/issn\/0092-8674","issn-type":"print"},{"issue":"18","key":"142_34626544","doi-asserted-by":"publisher","first-page":"7607","DOI":"10.1073\/pnas.0900688106","volume":"106","year":"2009","journal-title":"PNAS","ISSN":"https:\/\/id.crossref.org\/issn\/0027-8424","issn-type":"print"}],"container-title":["Brain"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/academic.oup.com\/brain\/article-pdf\/133\/4\/957\/870845\/awq002.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,10,22]],"date-time":"2021-10-22T20:38:18Z","timestamp":1634935098000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/brain\/article-lookup\/doi\/10.1093\/brain\/awq002"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2010,2,11]]},"references-count":141,"journal-issue":{"issue":"4","published-online":{"date-parts":[[2010,4,7]]},"published-print":{"date-parts":[[2010,4,1]]}},"URL":"https:\/\/doi.org\/10.1093\/brain\/awq002","relation":{},"ISSN":["0006-8950","1460-2156"],"issn-type":[{"value":"0006-8950","type":"print"},{"value":"1460-2156","type":"electronic"}],"subject":[],"published":{"date-parts":[[2010,2,11]]}}}