{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,20]],"date-time":"2026-03-20T02:15:40Z","timestamp":1773972940651,"version":"3.50.1"},"reference-count":46,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,10,15]],"date-time":"2025-10-15T00:00:00Z","timestamp":1760486400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000050","name":"National Heart Lung and Blood Institute","doi-asserted-by":"publisher","award":["R01 HL134321"],"award-info":[{"award-number":["R01 HL134321"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000050","name":"National Heart Lung and Blood Institute","doi-asserted-by":"publisher","award":["R01 HL123326"],"award-info":[{"award-number":["R01 HL123326"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000050","name":"National Heart Lung and Blood Institute","doi-asserted-by":"publisher","award":["N01 75N92019D00041"],"award-info":[{"award-number":["N01 75N92019D00041"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000050","name":"National Heart Lung and Blood Institute","doi-asserted-by":"publisher","award":["R35 HL150698"],"award-info":[{"award-number":["R35 HL150698"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Blood Vessels, Thrombosis & Hemostasis"],"published-print":{"date-parts":[[2026,2]]},"DOI":"10.1016\/j.bvth.2025.100119","type":"journal-article","created":{"date-parts":[[2025,10,25]],"date-time":"2025-10-25T07:30:01Z","timestamp":1761377401000},"page":"100119","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Treatment of canine hemophilia A via intraosseous delivery of a platelet-specific factor VIII\u2013lentiviral vector"],"prefix":"10.1016","volume":"3","author":[{"given":"Cameron W.","family":"Rementer","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chong","family":"Li","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8650-7096","authenticated-orcid":false,"given":"Timothy C.","family":"Nichols","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xiaohe","family":"Cai","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julia","family":"Joo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8842-610X","authenticated-orcid":false,"given":"Xuefeng","family":"Wang","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-1101-3820","authenticated-orcid":false,"given":"Mark","family":"Kloos","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nneka","family":"George","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ting-Yen","family":"Chao","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yuanyi","family":"Xu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mortimer","family":"Poncz","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hans D.","family":"Ochs","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0810-1776","authenticated-orcid":false,"given":"David J.","family":"Rawlings","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6520-2373","authenticated-orcid":false,"given":"Carol H.","family":"Miao","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"issue":"22","key":"10.1016\/j.bvth.2025.100119_bib1","doi-asserted-by":"crossref","first-page":"4449","DOI":"10.1182\/blood-2013-01-478669","article-title":"Progress toward inducing immunologic tolerance to factor VIII","volume":"121","author":"Scott","year":"2013","journal-title":"Blood"},{"issue":"1","key":"10.1016\/j.bvth.2025.100119_bib2","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1177\/2040620712464509","article-title":"Factor VIII inhibitors in hemophilia A: rationale and latest evidence","volume":"4","author":"Witmer","year":"2013","journal-title":"Ther Adv Hematol"},{"issue":"2 suppl 4","key":"10.1016\/j.bvth.2025.100119_bib3","first-page":"1","article-title":"Factor VIII inhibitors: structure and function in autoantibody and hemophilia A patients","volume":"31","author":"Hoyer","year":"1994","journal-title":"Semin Hematol"},{"issue":"suppl 5","key":"10.1016\/j.bvth.2025.100119_bib4","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1111\/hae.14102","article-title":"The societal burden of haemophilia A. I - A snapshot of haemophilia A in Australia and beyond","volume":"26","author":"Brown","year":"2020","journal-title":"Haemophilia"},{"issue":"5-6","key":"10.1016\/j.bvth.2025.100119_bib5","first-page":"322","article-title":"Hemophilia : a disease on the move","volume":"75","author":"P\u00e9ters","year":"2020","journal-title":"Rev Med Liege"},{"issue":"14","key":"10.1016\/j.bvth.2025.100119_bib6","doi-asserted-by":"crossref","first-page":"7647","DOI":"10.3390\/ijms22147647","article-title":"Gene therapy in hemophilia: recent advances","volume":"22","author":"Rodr\u00edguez-Merch\u00e1n","year":"2021","journal-title":"Int J Mol Sci"},{"issue":"6372","key":"10.1016\/j.bvth.2025.100119_bib7","doi-asserted-by":"crossref","DOI":"10.1126\/science.aan4672","article-title":"Gene therapy comes of age","volume":"359","author":"Dunbar","year":"2018","journal-title":"Science"},{"issue":"6304","key":"10.1016\/j.bvth.2025.100119_bib8","doi-asserted-by":"crossref","first-page":"1101","DOI":"10.1126\/science.aah6192","article-title":"Lentiviral vectors, two decades later","volume":"353","author":"Naldini","year":"2016","journal-title":"Science"},{"issue":"11","key":"10.1016\/j.bvth.2025.100119_bib9","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1182\/blood.2019003777","article-title":"Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues","volume":"138","author":"Leebeek","year":"2021","journal-title":"Blood"},{"issue":"4","key":"10.1016\/j.bvth.2025.100119_bib10","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1038\/mt.2015.20","article-title":"Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A","volume":"23","author":"Wang","year":"2015","journal-title":"Mol Ther"},{"issue":"5 pt 1","key":"10.1016\/j.bvth.2025.100119_bib11","doi-asserted-by":"crossref","first-page":"623","DOI":"10.1016\/S1525-0016(03)00073-X","article-title":"Efficient production of human FVIII in hemophilic mice using lentiviral vectors","volume":"7","author":"Kootstra","year":"2003","journal-title":"Mol Ther"},{"key":"10.1016\/j.bvth.2025.100119_bib12","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1016\/j.omtm.2018.01.011","article-title":"Platelet-targeted gene therapy for hemophilia","volume":"9","author":"Shi","year":"2018","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"suppl 1","key":"10.1016\/j.bvth.2025.100119_bib13","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1186\/s12959-016-0105-1","article-title":"Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors","volume":"14","author":"Miao","year":"2016","journal-title":"Thromb J"},{"issue":"4","key":"10.1016\/j.bvth.2025.100119_bib14","doi-asserted-by":"crossref","first-page":"136","DOI":"10.4252\/wjsc.v8.i4.136","article-title":"Migration of bone marrow progenitor cells in the adult brain of rats and rabbits","volume":"8","author":"Dennie","year":"2016","journal-title":"World J Stem Cells"},{"issue":"4","key":"10.1016\/j.bvth.2025.100119_bib15","doi-asserted-by":"crossref","first-page":"514","DOI":"10.1016\/j.ymthe.2006.05.014","article-title":"In vivo gene transfer into adult stem cells in unconditioned mice by in situ delivery of a lentiviral vector","volume":"14","author":"Worsham","year":"2006","journal-title":"Mol Ther"},{"issue":"22","key":"10.1016\/j.bvth.2025.100119_bib16","first-page":"5722","article-title":"Enhancing therapeutic efficacy of in vivo platelet-targeted gene therapy in hemophilia A mice","volume":"4","author":"Wang","year":"2020","journal-title":"Blood Adv"},{"issue":"5","key":"10.1016\/j.bvth.2025.100119_bib17","doi-asserted-by":"crossref","first-page":"894","DOI":"10.1111\/jth.13301","article-title":"Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies","volume":"14","author":"Nichols","year":"2016","journal-title":"J Thromb Haemost"},{"issue":"1","key":"10.1016\/j.bvth.2025.100119_bib18","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1016\/j.ymthe.2004.04.009","article-title":"Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice","volume":"10","author":"Ye","year":"2004","journal-title":"Mol Ther"},{"issue":"3","key":"10.1016\/j.bvth.2025.100119_bib19","doi-asserted-by":"crossref","first-page":"538","DOI":"10.1007\/s12010-008-8381-2","article-title":"Polyethylenimine-based transfection method as a simple and effective way to produce recombinant lentiviral vectors","volume":"157","author":"Toledo","year":"2009","journal-title":"Appl Biochem Biotechnol"},{"issue":"19","key":"10.1016\/j.bvth.2025.100119_bib20","doi-asserted-by":"crossref","first-page":"5556","DOI":"10.1182\/bloodadvances.2022008079","article-title":"Intraosseous delivery of platelet-targeted factor VIII lentiviral vector in humanized NBSGW mice","volume":"6","author":"Joo","year":"2022","journal-title":"Blood Adv"},{"issue":"5","key":"10.1016\/j.bvth.2025.100119_bib21","doi-asserted-by":"crossref","first-page":"e963","DOI":"10.1111\/j.1365-2516.2011.02580.x","article-title":"Pharmacokinetics and ex vivo whole blood clot formation of a new recombinant FVIII (N8) in haemophilia A dogs","volume":"17","author":"Karpf","year":"2011","journal-title":"Haemophilia"},{"issue":"3","key":"10.1016\/j.bvth.2025.100119_bib22","doi-asserted-by":"crossref","first-page":"474","DOI":"10.1111\/j.1538-7836.2011.04613.x","article-title":"Sensitivity of whole blood clotting time and activated partial thromboplastin time for factor IX: relevance to gene therapy and determination of post-transfusion elimination time of canine factor IX in hemophilia B dogs","volume":"10","author":"Nichols","year":"2012","journal-title":"J Thromb Haemost"},{"issue":"suppl 4","key":"10.1016\/j.bvth.2025.100119_bib23","doi-asserted-by":"crossref","first-page":"S42","DOI":"10.1016\/S0020-1383(14)70009-9","article-title":"Bone marrow mesenchymal stem cell aspirates from alternative sources: is the knee as good as the iliac crest?","volume":"45","author":"Narbona-Carceles","year":"2014","journal-title":"Injury"},{"issue":"12","key":"10.1016\/j.bvth.2025.100119_bib24","doi-asserted-by":"crossref","first-page":"4006","DOI":"10.1182\/blood-2003-05-1519","article-title":"Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment","volume":"102","author":"Yarovoi","year":"2003","journal-title":"Blood"},{"issue":"6","key":"10.1016\/j.bvth.2025.100119_bib25","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1111\/j.1365-2516.2012.02896.x","article-title":"Pharmacokinetics and pharmacodynamics of turoctocog alfa and N8-GP in haemophilia A dogs","volume":"18","author":"Agers\u00f8","year":"2012","journal-title":"Haemophilia"},{"issue":"13","key":"10.1016\/j.bvth.2025.100119_bib26","doi-asserted-by":"crossref","first-page":"3024","DOI":"10.1182\/blood-2011-08-367813","article-title":"Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs","volume":"119","author":"Dumont","year":"2012","journal-title":"Blood"},{"issue":"2","key":"10.1016\/j.bvth.2025.100119_bib27","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1161\/01.RES.88.2.137","article-title":"Gene transfer as an approach to treating hemophilia","volume":"88","author":"High","year":"2001","journal-title":"Circ Res"},{"issue":"6","key":"10.1016\/j.bvth.2025.100119_bib28","doi-asserted-by":"crossref","first-page":"550","DOI":"10.2174\/1566523215666150929110903","article-title":"Lentiviral-mediated gene therapy in Fanconi anemia-a mice reveals long-term engraftment and continuous turnover of corrected HSCs","volume":"15","author":"Molina-Estevez","year":"2015","journal-title":"Curr Gene Ther"},{"issue":"2","key":"10.1016\/j.bvth.2025.100119_bib29","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1111\/j.1538-7836.2007.02346.x","article-title":"Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A","volume":"5","author":"Shi","year":"2007","journal-title":"J Thromb Haemost"},{"issue":"8","key":"10.1016\/j.bvth.2025.100119_bib30","doi-asserted-by":"crossref","first-page":"2855","DOI":"10.1182\/blood-2007-04-082602","article-title":"Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens","volume":"110","author":"Ide","year":"2007","journal-title":"Blood"},{"issue":"4","key":"10.1016\/j.bvth.2025.100119_bib31","first-page":"314","article-title":"Manufacturing cell and gene therapies: challenges in clinical translation","volume":"44","author":"Lee","year":"2024","journal-title":"alm"},{"issue":"6","key":"10.1016\/j.bvth.2025.100119_bib32","first-page":"e62817","article-title":"Improving hemophilia care in low- and middle-income countries: addressing challenges and enhancing quality of life","volume":"16","author":"Perolla","year":"2024","journal-title":"Cureus"},{"issue":"9","key":"10.1016\/j.bvth.2025.100119_bib33","doi-asserted-by":"crossref","first-page":"e689","DOI":"10.1016\/S2352-3026(22)00209-5","article-title":"Achieving access to haemophilia care in low-income and lower-middle-income countries: expanded Humanitarian Aid Program of the World Federation of Hemophilia after 5 years","volume":"9","author":"Pierce","year":"2022","journal-title":"Lancet Haematol"},{"issue":"5","key":"10.1016\/j.bvth.2025.100119_bib34","doi-asserted-by":"crossref","first-page":"2015","DOI":"10.1016\/j.ymthe.2025.03.043","article-title":"Gene therapy for hemophilia - from basic science to first approvals of \"one-and-done\" therapies","volume":"33","author":"Herzog","year":"2025","journal-title":"Mol Ther"},{"issue":"1","key":"10.1016\/j.bvth.2025.100119_bib35","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1182\/blood-2013-01-306647","article-title":"Immune responses to AAV vectors: overcoming barriers to successful gene therapy","volume":"122","author":"Mingozzi","year":"2013","journal-title":"Blood"},{"issue":"8","key":"10.1016\/j.bvth.2025.100119_bib36","doi-asserted-by":"crossref","first-page":"694","DOI":"10.1056\/NEJMoa2211075","article-title":"Two-year outcomes of valoctocogene roxaparvovec therapy for hemophilia A","volume":"388","author":"Mahlangu","year":"2023","journal-title":"N Engl J Med"},{"issue":"5","key":"10.1016\/j.bvth.2025.100119_bib37","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1056\/NEJMoa2410597","article-title":"Lentiviral gene therapy with CD34+ hematopoietic cells for hemophilia A","volume":"392","author":"Srivastava","year":"2025","journal-title":"N Engl J Med"},{"issue":"4","key":"10.1016\/j.bvth.2025.100119_bib38","doi-asserted-by":"crossref","first-page":"412","DOI":"10.1056\/NEJMc2415164","article-title":"Platelet-targeted gene therapy for hemophilia A with inhibitor history","volume":"392","author":"Eapen","year":"2025","journal-title":"N Engl J Med"},{"key":"10.1016\/j.bvth.2025.100119_bib39","doi-asserted-by":"crossref","first-page":"494","DOI":"10.3389\/fimmu.2020.00494","article-title":"The immune response to the FVIII gene therapy in preclinical models","volume":"11","author":"Patel","year":"2020","journal-title":"Front Immunol"},{"issue":"1","key":"10.1016\/j.bvth.2025.100119_bib40","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1016\/S1096-7192(03)00049-0","article-title":"Expression of human factor VIII under control of the platelet-specific alphaIIb promoter in megakaryocytic cell line as well as storage together with VWF","volume":"79","author":"Shi","year":"2003","journal-title":"Mol Genet Metab"},{"issue":"7","key":"10.1016\/j.bvth.2025.100119_bib41","doi-asserted-by":"crossref","first-page":"1160","DOI":"10.1111\/j.1538-7836.2008.02992.x","article-title":"Platelet-delivered factor VIII provides limited resistance to anti-factor VIII inhibitors","volume":"6","author":"Gewirtz","year":"2008","journal-title":"J Thromb Haemost"},{"issue":"7","key":"10.1016\/j.bvth.2025.100119_bib42","doi-asserted-by":"crossref","first-page":"1791","DOI":"10.3324\/haematol.2019.230771","article-title":"International recommendations on the diagnosis and treatment of acquired hemophilia A","volume":"105","author":"Tiede","year":"2020","journal-title":"Haematologica"},{"issue":"7","key":"10.1016\/j.bvth.2025.100119_bib43","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1002\/ajh.24777","article-title":"Acquired hemophilia A: updated review of evidence and treatment guidance","volume":"92","author":"Kruse-Jarres","year":"2017","journal-title":"Am J Hematol"},{"issue":"10","key":"10.1016\/j.bvth.2025.100119_bib44","doi-asserted-by":"crossref","first-page":"2644","DOI":"10.1182\/blood.V81.10.2644.2644","article-title":"The roles of von Willebrand factor and factor VIII in arterial thrombosis: studies in canine von Willebrand disease and hemophilia A","volume":"81","author":"Nichols","year":"1993","journal-title":"Blood"},{"issue":"1","key":"10.1016\/j.bvth.2025.100119_bib45","doi-asserted-by":"crossref","first-page":"145","DOI":"10.1182\/blood-2004-02-0464","article-title":"Re-establishment of VWF-dependent Weibel-Palade bodies in VWD endothelial cells","volume":"105","author":"Haberichter","year":"2005","journal-title":"Blood"},{"issue":"6","key":"10.1016\/j.bvth.2025.100119_bib46","article-title":"The factor VIII\/von Willebrand factor complex: basic and clinical issues","volume":"88","author":"Federici","year":"2003","journal-title":"Haematologica"}],"container-title":["Blood Vessels, Thrombosis & Hemostasis"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2950327225000762?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2950327225000762?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,3,20]],"date-time":"2026-03-20T01:28:33Z","timestamp":1773970113000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2950327225000762"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,2]]},"references-count":46,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,2]]}},"alternative-id":["S2950327225000762"],"URL":"https:\/\/doi.org\/10.1016\/j.bvth.2025.100119","relation":{},"ISSN":["2950-3272"],"issn-type":[{"value":"2950-3272","type":"print"}],"subject":[],"published":{"date-parts":[[2026,2]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Treatment of canine hemophilia A via intraosseous delivery of a platelet-specific factor VIII\u2013lentiviral vector","name":"articletitle","label":"Article Title"},{"value":"Blood Vessels, Thrombosis & Hemostasis","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.bvth.2025.100119","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 American Society of Hematology. Published by Elsevier Inc. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.","name":"copyright","label":"Copyright"}],"article-number":"100119"}}