{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T18:41:20Z","timestamp":1791398480827,"version":"4.3.3"},"reference-count":28,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2016,3,1]],"date-time":"2016-03-01T00:00:00Z","timestamp":1456790400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81400101"],"award-info":[{"award-number":["81400101"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["31571313"],"award-info":[{"award-number":["31571313"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81470299"],"award-info":[{"award-number":["81470299"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81272540"],"award-info":[{"award-number":["81272540"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81271944"],"award-info":[{"award-number":["81271944"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012166","name":"National Basic Research Program of China","doi-asserted-by":"crossref","award":["2010CB529903"],"award-info":[{"award-number":["2010CB529903"]}],"id":[{"id":"10.13039\/501100012166","id-type":"DOI","asserted-by":"crossref"}]},{"name":"China Postdoctoral Science Foundation funded project","award":["2015M580697"],"award-info":[{"award-number":["2015M580697"]}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Biochemical and Biophysical Research Communications"],"published-print":{"date-parts":[[2016,3]]},"DOI":"10.1016\/j.bbrc.2016.02.083","type":"journal-article","created":{"date-parts":[[2016,2,25]],"date-time":"2016-02-25T08:56:24Z","timestamp":1456390584000},"page":"144-149","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":31,"title":["Targeting of the human F8 at the multicopy rDNA locus in Hemophilia a patient-derived iPSCs using TALENickases"],"prefix":"10.1016","volume":"472","author":[{"given":"Jialun","family":"Pang","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yong","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhuo","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhiqing","family":"Hu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaolin","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xuyun","family":"Hu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoyan","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xionghao","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miaojin","family":"Zhou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bo","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanchi","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mai","family":"Feng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Desheng","family":"Liang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.bbrc.2016.02.083_bib1","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1179\/1024533214Z.000000000262","article-title":"Haematology clinic: haemophilia A","volume":"19","author":"Mannucci","year":"2014","journal-title":"Hematology"},{"key":"10.1016\/j.bbrc.2016.02.083_bib2","doi-asserted-by":"crossref","first-page":"1954","DOI":"10.1111\/jth.12750","article-title":"Progress and challenges in the development of a cell-based therapy for hemophilia A","volume":"12","author":"Fomin","year":"2014","journal-title":"J.\u00a0Thromb. Haemost."},{"key":"10.1016\/j.bbrc.2016.02.083_bib3","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1038\/gt.2012.76","article-title":"Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A","volume":"20","author":"Johnston","year":"2013","journal-title":"Gene Ther."},{"key":"10.1016\/j.bbrc.2016.02.083_bib4","doi-asserted-by":"crossref","first-page":"2773","DOI":"10.1038\/ncomms3773","article-title":"Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A","volume":"4","author":"Du","year":"2013","journal-title":"Nat. Commun."},{"key":"10.1016\/j.bbrc.2016.02.083_bib5","doi-asserted-by":"crossref","first-page":"1034","DOI":"10.1016\/j.ymthe.2005.09.007","article-title":"Correction of murine hemophilia A by hematopoietic stem cell gene therapy","volume":"12","author":"Moayeri","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.bbrc.2016.02.083_bib6","doi-asserted-by":"crossref","first-page":"556","DOI":"10.2741\/4324","article-title":"Gene therapy for hemophilia","volume":"20","author":"Rogers","year":"2015","journal-title":"Front. Biosci. (Landmark Ed)"},{"key":"10.1016\/j.bbrc.2016.02.083_bib7","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/j.stem.2015.07.001","article-title":"Functional correction of large factor VIII Gene chromosomal inversions in hemophilia A patient-derived iPSCs using CRISPR-Cas9","volume":"17","author":"Park","year":"2015","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.bbrc.2016.02.083_bib8","doi-asserted-by":"crossref","first-page":"1777","DOI":"10.1182\/blood-2014-12-615492","article-title":"In\u00a0vivo genome editing of the albumin locus as a platform for protein replacement therapy","volume":"126","author":"Sharma","year":"2015","journal-title":"Blood"},{"key":"10.1016\/j.bbrc.2016.02.083_bib9","doi-asserted-by":"crossref","first-page":"9253","DOI":"10.1073\/pnas.1323941111","article-title":"Targeted inversion and reversion of the blood coagulation factor 8 gene in human iPS cells using TALENs","volume":"111","author":"Park","year":"2014","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.bbrc.2016.02.083_bib10","doi-asserted-by":"crossref","first-page":"e37071","DOI":"10.1371\/journal.pone.0037071","article-title":"Targeting of the human coagulation factor IX gene at rDNA locus of human embryonic stem cells","volume":"7","author":"Liu","year":"2012","journal-title":"PLoS One"},{"key":"10.1016\/j.bbrc.2016.02.083_bib11","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1111\/j.1538-7836.2007.02355.x","article-title":"Non-viral ex\u00a0vivo transduction of human hepatocyte cells to express factor VIII using a human ribosomal DNA-targeting vector","volume":"5","author":"Liu","year":"2007","journal-title":"J.\u00a0Thromb. Haemost."},{"key":"10.1016\/j.bbrc.2016.02.083_bib12","doi-asserted-by":"crossref","first-page":"1053","DOI":"10.1093\/abbs\/gmp080","article-title":"A\u00a0non-viral vector for potential DMD gene therapy study by targeting a minidystrophin-GFP fusion gene into the hrDNA locus","volume":"41","author":"Yang","year":"2009","journal-title":"Acta Biochim. Biophys. Sin. (Shanghai)"},{"key":"10.1016\/j.bbrc.2016.02.083_bib13","doi-asserted-by":"crossref","first-page":"135189","DOI":"10.1155\/2013\/135189","article-title":"Nonviral gene targeting at rDNA locus of human mesenchymal stem cells","volume":"2013","author":"Hu","year":"2013","journal-title":"Biomed. Res. Int."},{"key":"10.1016\/j.bbrc.2016.02.083_bib14","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1101\/gr.6858507","article-title":"Genomic architecture and inheritance of human ribosomal RNA gene clusters","volume":"18","author":"Stults","year":"2008","journal-title":"Genome Res."},{"key":"10.1016\/j.bbrc.2016.02.083_bib15","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1007\/BF00286654","article-title":"The genetic significance of accessory bisatellited marker chromosomes","volume":"65","author":"Steinbach","year":"1983","journal-title":"Hum. Genet."},{"key":"10.1016\/j.bbrc.2016.02.083_bib16","doi-asserted-by":"crossref","first-page":"19","DOI":"10.32607\/20758251-2014-6-3-19-40","article-title":"TALEN and CRISPR\/Cas genome editing systems: tools of discovery","volume":"6","author":"Nemudryi","year":"2014","journal-title":"Acta Naturae"},{"key":"10.1016\/j.bbrc.2016.02.083_bib17","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1038\/nbt.1927","article-title":"Genetic engineering of human pluripotent cells using TALE nucleases","volume":"29","author":"Hockemeyer","year":"2011","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.bbrc.2016.02.083_bib18","doi-asserted-by":"crossref","first-page":"5099","DOI":"10.1073\/pnas.0810588106","article-title":"Generation of a nicking enzyme that stimulates site-specific gene conversion from the I-AniI LAGLIDADG homing endonuclease","volume":"106","author":"McConnell Smith","year":"2009","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.bbrc.2016.02.083_bib19","doi-asserted-by":"crossref","first-page":"261","DOI":"10.1016\/j.bbrc.2014.02.099","article-title":"TALE nickase mediates high efficient targeted transgene integration at the human multi-copy ribosomal DNA locus","volume":"446","author":"Wu","year":"2014","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.bbrc.2016.02.083_bib20","doi-asserted-by":"crossref","first-page":"4902","DOI":"10.1182\/blood-2009-07-232546","article-title":"Activation of human endothelial cells from specific vascular beds induces the release of a FVIII storage pool","volume":"115","author":"Shahani","year":"2010","journal-title":"Blood"},{"key":"10.1016\/j.bbrc.2016.02.083_bib21","doi-asserted-by":"crossref","first-page":"e107","DOI":"10.1093\/nar\/gkr409","article-title":"Efficient recombinase-mediated cassette exchange at the AAVS1 locus in human embryonic stem cells using baculoviral vectors","volume":"39","author":"Ramachandra","year":"2011","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.bbrc.2016.02.083_bib22","first-page":"1341","article-title":"Cellular localization of the chemokine receptor CCR5. Correlation to cellular targets of HIV-1 infection","volume":"151","author":"Rottman","year":"1997","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.bbrc.2016.02.083_bib23","doi-asserted-by":"crossref","first-page":"1477","DOI":"10.1038\/nbt1362","article-title":"Identification and targeting of the ROSA26 locus in human embryonic stem cells","volume":"25","author":"Irion","year":"2007","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.bbrc.2016.02.083_bib24","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1038\/nrc3179","article-title":"Safe harbours for the integration of new DNA in the human genome","volume":"12","author":"Sadelain","year":"2012","journal-title":"Nat. Rev. Cancer"},{"key":"10.1016\/j.bbrc.2016.02.083_bib25","doi-asserted-by":"crossref","first-page":"1053","DOI":"10.1089\/scd.2014.0347","article-title":"Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR\/Cas9 system","volume":"24","author":"Song","year":"2015","journal-title":"Stem Cells Dev."},{"key":"10.1016\/j.bbrc.2016.02.083_bib26","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/j.stemcr.2014.10.013","article-title":"Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9","volume":"4","author":"Li","year":"2015","journal-title":"Stem Cell Rep."},{"key":"10.1016\/j.bbrc.2016.02.083_bib27","doi-asserted-by":"crossref","first-page":"1318","DOI":"10.1038\/nm.3270","article-title":"Endogenous factor VIII synthesis from the intron 22-inverted F8 locus may modulate the immunogenicity of replacement therapy for hemophilia A","volume":"19","author":"Pandey","year":"2013","journal-title":"Nat. Med."},{"key":"10.1016\/j.bbrc.2016.02.083_bib28","doi-asserted-by":"crossref","first-page":"1101","DOI":"10.1182\/blood-2008-04-152959","article-title":"Analysis of the spatial and temporal characteristics of platelet-delivered factor VIII-based clots","volume":"112","author":"Neyman","year":"2008","journal-title":"Blood"}],"container-title":["Biochemical and Biophysical Research Communications"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0006291X16302728?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0006291X16302728?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2020,5,16]],"date-time":"2020-05-16T14:14:25Z","timestamp":1589638465000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0006291X16302728"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,3]]},"references-count":28,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2016,3]]}},"alternative-id":["S0006291X16302728"],"URL":"https:\/\/doi.org\/10.1016\/j.bbrc.2016.02.083","relation":{},"ISSN":["0006-291X"],"issn-type":[{"value":"0006-291X","type":"print"}],"subject":[],"published":{"date-parts":[[2016,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Targeting of the human F8 at the multicopy rDNA locus in Hemophilia a patient-derived iPSCs using TALENickases","name":"articletitle","label":"Article Title"},{"value":"Biochemical and Biophysical Research Communications","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.bbrc.2016.02.083","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2016 Elsevier Inc. All rights reserved.","name":"copyright","label":"Copyright"}]}}