{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,2]],"date-time":"2026-09-02T15:12:23Z","timestamp":1788361943725,"version":"build-2803163510"},"reference-count":116,"publisher":"SAGE Publications","issue":"17-18","license":[{"start":{"date-parts":[[2023,9,1]],"date-time":"2023-09-01T00:00:00Z","timestamp":1693526400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/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":[[2023,9,1]]},"DOI":"10.1089\/hum.2023.112","type":"journal-article","created":{"date-parts":[[2023,9,6]],"date-time":"2023-09-06T13:24:18Z","timestamp":1694006658000},"page":"782-792","source":"Crossref","is-referenced-by-count":45,"title":["Hemophilia Gene Therapy: The End of the Beginning?"],"prefix":"10.1177","volume":"34","author":[{"given":"Dries","family":"De Wolf","sequence":"first","affiliation":[{"name":"Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel, Brussels, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kshitiz","family":"Singh","sequence":"additional","affiliation":[{"name":"Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel, Brussels, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marinee K.","family":"Chuah","sequence":"additional","affiliation":[{"name":"Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel, Brussels, Belgium"},{"name":"Department of Cardiovascular Sciences, Center for Molecular and Vascular Biology, University of Leuven, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4230-745X","authenticated-orcid":false,"given":"Thierry","family":"VandenDriessche","sequence":"additional","affiliation":[{"name":"Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel, Brussels, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","reference":[{"key":"B1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(15)01123-X"},{"key":"B2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2015-01-528414"},{"key":"B3","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMe1707802"},{"key":"B4","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(22)00300-3"},{"key":"B5","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2516.2002.00626.x"},{"key":"B6","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13494"},{"key":"B7","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2008.71"},{"key":"B8","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-05-423210"},{"key":"B9","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2004.00926.x"},{"key":"B10","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-08-520478"},{"key":"B11","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2018.137"},{"key":"B12","doi-asserted-by":"publisher","DOI":"10.1038\/ncomms3773"},{"key":"B13","doi-asserted-by":"publisher","DOI":"10.1126\/science.1068893"},{"key":"B14","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200301163480314"},{"key":"B15","doi-asserted-by":"publisher","DOI":"10.1172\/JCI37630"},{"key":"B16","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2014.3"},{"key":"B17","doi-asserted-by":"publisher","DOI":"10.1128\/JVI.72.11.8463-8471.1998"},{"key":"B18","doi-asserted-by":"publisher","DOI":"10.1086\/595830"},{"key":"B19","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2009.182"},{"key":"B20","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2021.287"},{"key":"B21","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2020.06.001"},{"key":"B22","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2021.658399"},{"key":"B23","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-med-043021-033013"},{"key":"B24","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-07-820720"},{"key":"B25","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2017.03.033"},{"key":"B26","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1407309"},{"key":"B27","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1708538"},{"key":"B28","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1708483"},{"key":"B29","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1908490"},{"key":"B30","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2104205"},{"issue":"1","key":"B31","first-page":"604","volume":"130","author":"George LA","year":"2017","journal-title":"Blood"},{"key":"B32","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2022-159421"},{"issue":"1","key":"B33","first-page":"OC","volume":"4","author":"Pipe SW","year":"2020","journal-title":"Res Pract Thromb Haemost"},{"key":"B34","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2010-05-282707"},{"key":"B35","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.26018"},{"key":"B36","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-10-3591"},{"key":"B37","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-02-554501"},{"key":"B38","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-02-555151"},{"key":"B39","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12412"},{"key":"B40","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.274.28.19587"},{"key":"B41","doi-asserted-by":"publisher","DOI":"10.1038\/317726a0"},{"key":"B42","doi-asserted-by":"publisher","DOI":"10.1038\/ng1153"},{"key":"B43","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2020.07.005"},{"key":"B44","doi-asserted-by":"publisher","DOI":"10.1038\/mtm.2016.64"},{"key":"B45","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019002867"},{"key":"B46","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2113708"},{"key":"B47","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2017.12.009"},{"key":"B48","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2211075"},{"key":"B50","doi-asserted-by":"publisher","DOI":"10.1038\/s41587-020-0741-7"},{"key":"B51","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2021014735"},{"key":"B52","doi-asserted-by":"publisher","DOI":"10.1038\/nm1549"},{"key":"B53","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMe2212347"},{"key":"B54","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020005683"},{"key":"B55","doi-asserted-by":"publisher","DOI":"10.1038\/nm1358"},{"key":"B56","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1108046"},{"key":"B57","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2017-09-804419"},{"key":"B58","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa0904377"},{"key":"B59","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-05-432591"},{"key":"B60","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-06-440123"},{"key":"B61","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-10-534032"},{"key":"B62","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-07-588194"},{"key":"B63","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2014.106"},{"key":"B64","doi-asserted-by":"publisher","DOI":"10.1160\/TH13-02-0154"},{"key":"B65","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2211644"},{"issue":"4","key":"B66","volume":"30","author":"Pipe SW","year":"2022","journal-title":"Mol Ther"},{"key":"B69","volume-title":"Long-Term Data from Phase 3 Studies on FIX Gene Therapy","author":"Meisbach W.","year":"2023"},{"key":"B71","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-03-080093"},{"key":"B72","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15281"},{"key":"B73","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2017.11.007"},{"key":"B74","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2022.08.002"},{"key":"B75","doi-asserted-by":"publisher","DOI":"10.1002\/jgm.1343"},{"key":"B76","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2018.02.023"},{"key":"B77","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.18.10379"},{"key":"B78","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-022-30102-3"},{"key":"B79","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aav7325"},{"key":"B80","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aaa1405"},{"key":"B81","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2016.38"},{"key":"B82","doi-asserted-by":"publisher","DOI":"10.1038\/nature10177"},{"key":"B83","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-04-497354"},{"key":"B85","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2022.10.010"},{"key":"B86","doi-asserted-by":"publisher","DOI":"10.15252\/emmm.201506039"},{"key":"B87","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-017-04625-5"},{"key":"B88","doi-asserted-by":"publisher","DOI":"10.1007\/s00439-017-1801-z"},{"key":"B89","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019000790"},{"key":"B90","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-019-53198-y"},{"key":"B91","doi-asserted-by":"publisher","DOI":"10.1186\/s13059-019-1907-9"},{"key":"B92","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2020.06.025"},{"key":"B93","doi-asserted-by":"publisher","DOI":"10.3389\/fcell.2021.672564"},{"key":"B94","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtn.2022.08.002"},{"key":"B95","unstructured":"95. Hon-Ren Huang. CRISPR\/Cas9-mediated targeted insertion of human F9 achieves therapeutic circulating protein levels in mice and non-human primates. Presented at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy, April 29, 2019."},{"key":"B96","doi-asserted-by":"publisher","DOI":"10.1038\/ng1380"},{"key":"B97","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-019-12449-2"},{"key":"B98","doi-asserted-by":"publisher","DOI":"10.1186\/s12864-020-6655-4"},{"key":"B99","doi-asserted-by":"publisher","DOI":"10.1038\/nature13864"},{"key":"B100","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019004625"},{"key":"B101","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-02-073304"},{"issue":"1","key":"B102","first-page":"88","volume":"7","author":"Coa O","year":"2009","journal-title":"J Thromb Haemostasis"},{"key":"B103","doi-asserted-by":"publisher","DOI":"10.1002\/emmm.201302859"},{"key":"B104","doi-asserted-by":"publisher","DOI":"10.1002\/hep.24230"},{"key":"B105","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2011.33"},{"key":"B106","doi-asserted-by":"publisher","DOI":"10.1189\/jlb.2AB0613-342RR"},{"key":"B107","doi-asserted-by":"publisher","DOI":"10.1016\/j.jhep.2014.04.027"},{"key":"B108","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2013.94"},{"key":"B109","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2017.02.014"},{"key":"B110","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2020.00638"},{"key":"B111","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2021.258"},{"key":"B112","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2020.01293"},{"key":"B113","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020006005"},{"key":"B114","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2022.07.005"},{"key":"B115","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-022-01751-0"},{"key":"B116","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2022.11.001"},{"key":"B117","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2022.11.006"},{"key":"B118","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2022.050"},{"key":"B119","doi-asserted-by":"publisher","DOI":"10.1038\/s41588-021-00838-7"},{"key":"B120","doi-asserted-by":"publisher","DOI":"10.1016\/j.stem.2022.09.001"},{"key":"B121","doi-asserted-by":"publisher","DOI":"10.1038\/s41434-021-00232-2"}],"container-title":["Human Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.liebertpub.com\/doi\/full-xml\/10.1089\/hum.2023.112","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.liebertpub.com\/doi\/pdf\/10.1089\/hum.2023.112","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T07:56:41Z","timestamp":1777449401000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/full\/10.1089\/hum.2023.112"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,9,1]]},"references-count":116,"journal-issue":{"issue":"17-18","published-print":{"date-parts":[[2023,9,1]]}},"alternative-id":["10.1089\/hum.2023.112"],"URL":"https:\/\/doi.org\/10.1089\/hum.2023.112","relation":{},"ISSN":["1043-0342","1557-7422"],"issn-type":[{"value":"1043-0342","type":"print"},{"value":"1557-7422","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,9,1]]}}}