{"id":"https://openalex.org/W4415540744","doi":"https://doi.org/10.1016/j.bvth.2025.100119","title":"Treatment of canine hemophilia A via intraosseous delivery of a platelet-specific factor VIII\u2013lentiviral vector","display_name":"Treatment of canine hemophilia A via intraosseous delivery of a platelet-specific factor VIII\u2013lentiviral vector","publication_year":2025,"publication_date":"2025-10-25","ids":{"openalex":"https://openalex.org/W4415540744","doi":"https://doi.org/10.1016/j.bvth.2025.100119","pmid":"https://pubmed.ncbi.nlm.nih.gov/41608029","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/12835592"},"language":"en","primary_location":{"id":"doi:10.1016/j.bvth.2025.100119","doi":"https://doi.org/10.1016/j.bvth.2025.100119","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.bvth.2025.100119","pdf_url":null,"source":{"id":"https://openalex.org/S5407037026","display_name":"Blood Vessels Thrombosis & Hemostasis","issn_l":"2950-3272","issn":["2950-3272"],"is_oa":true,"is_in_doaj":true,"is_core":false,"listed_in":["doaj","ki-jl-1"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood Vessels, Thrombosis &amp; Hemostasis","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","datacite","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://doi.org/10.1016/j.bvth.2025.100119","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5093245019","display_name":"Cameron W. Rementer","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4405262941","display_name":"Seattle Children's Research Institute","ror":"https://ror.org/00cz0md82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1295499534","https://openalex.org/I4405262941"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Cameron W. Rementer","raw_affiliation_strings":["Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","institution_ids":["https://openalex.org/I4405262941"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5100424944","display_name":"Chong Li","orcid":"https://orcid.org/0000-0002-9215-6779","observed_orcids":["https://orcid.org/0000-0002-9215-6779"]},"institutions":[{"id":"https://openalex.org/I4405262941","display_name":"Seattle Children's Research Institute","ror":"https://ror.org/00cz0md82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1295499534","https://openalex.org/I4405262941"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Chong Li","raw_affiliation_strings":["Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","institution_ids":["https://openalex.org/I4405262941"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5038704186","display_name":"Timothy C. Nichols","orcid":"https://orcid.org/0000-0001-8650-7096","observed_orcids":["https://orcid.org/0000-0001-8650-7096"]},"institutions":[{"id":"https://openalex.org/I114027177","display_name":"University of North Carolina at Chapel Hill","ror":"https://ror.org/0130frc33","country_code":"US","type":"education","lineage":["https://openalex.org/I114027177"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Timothy C. Nichols","raw_affiliation_strings":["Department of Pathology and Laboratory Medicine and Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill, NC"],"raw_orcid":"https://orcid.org/0000-0001-8650-7096","affiliations":[{"raw_affiliation_string":"Department of Pathology and Laboratory Medicine and Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill, NC","institution_ids":["https://openalex.org/I114027177"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5113778223","display_name":"Xiaohe Cai","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4405262941","display_name":"Seattle Children's Research Institute","ror":"https://ror.org/00cz0md82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1295499534","https://openalex.org/I4405262941"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Xiaohe Cai","raw_affiliation_strings":["Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","institution_ids":["https://openalex.org/I4405262941"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5015282610","display_name":"Julia H. Joo","orcid":"https://orcid.org/0009-0000-1299-8144","observed_orcids":["https://orcid.org/0009-0000-1299-8144"]},"institutions":[{"id":"https://openalex.org/I4405262941","display_name":"Seattle Children's Research Institute","ror":"https://ror.org/00cz0md82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1295499534","https://openalex.org/I4405262941"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Julia Joo","raw_affiliation_strings":["Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","institution_ids":["https://openalex.org/I4405262941"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5003736861","display_name":"Xuefeng Wang","orcid":"https://orcid.org/0000-0001-8842-610X","observed_orcids":["https://orcid.org/0000-0001-8842-610X"]},"institutions":[{"id":"https://openalex.org/I4405262941","display_name":"Seattle Children's Research Institute","ror":"https://ror.org/00cz0md82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1295499534","https://openalex.org/I4405262941"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Xuefeng Wang","raw_affiliation_strings":["Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA"],"raw_orcid":"https://orcid.org/0000-0001-8842-610X","affiliations":[{"raw_affiliation_string":"Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","institution_ids":["https://openalex.org/I4405262941"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5073293348","display_name":"Mark T. Kloos","orcid":"https://orcid.org/0009-0009-1101-3820","observed_orcids":["https://orcid.org/0009-0009-1101-3820"]},"institutions":[{"id":"https://openalex.org/I114027177","display_name":"University of North Carolina at Chapel Hill","ror":"https://ror.org/0130frc33","country_code":"US","type":"education","lineage":["https://openalex.org/I114027177"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Mark Kloos","raw_affiliation_strings":["Department of Pathology and Laboratory Medicine and Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill, NC"],"raw_orcid":"https://orcid.org/0009-0009-1101-3820","affiliations":[{"raw_affiliation_string":"Department of Pathology and Laboratory Medicine and Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill, NC","institution_ids":["https://openalex.org/I114027177"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Nneka George","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I114027177","display_name":"University of North Carolina at Chapel Hill","ror":"https://ror.org/0130frc33","country_code":"US","type":"education","lineage":["https://openalex.org/I114027177"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Nneka George","raw_affiliation_strings":["Department of Pathology and Laboratory Medicine and Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill, NC"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pathology and Laboratory Medicine and Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill, NC","institution_ids":["https://openalex.org/I114027177"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5020068553","display_name":"Ting-Yen Chao","orcid":"https://orcid.org/0009-0005-3452-9660","observed_orcids":["https://orcid.org/0009-0005-3452-9660"]},"institutions":[{"id":"https://openalex.org/I4405262941","display_name":"Seattle Children's Research Institute","ror":"https://ror.org/00cz0md82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1295499534","https://openalex.org/I4405262941"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Ting-Yen Chao","raw_affiliation_strings":["Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","institution_ids":["https://openalex.org/I4405262941"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5054079984","display_name":"Yuanyi Xu","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4405262941","display_name":"Seattle Children's Research Institute","ror":"https://ror.org/00cz0md82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1295499534","https://openalex.org/I4405262941"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Yuanyi Xu","raw_affiliation_strings":["Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","institution_ids":["https://openalex.org/I4405262941"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5030315782","display_name":"Mortimer Poncz","orcid":"https://orcid.org/0000-0001-7237-3613","observed_orcids":["https://orcid.org/0000-0001-7237-3613"]},"institutions":[{"id":"https://openalex.org/I1335321130","display_name":"Children's Hospital of Philadelphia","ror":"https://ror.org/01z7r7q48","country_code":"US","type":"funder","lineage":["https://openalex.org/I1335321130"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Mortimer Poncz","raw_affiliation_strings":["Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5062237763","display_name":"Hans D. Ochs","orcid":"https://orcid.org/0000-0002-7726-3218","observed_orcids":["https://orcid.org/0000-0002-7726-3218"]},"institutions":[{"id":"https://openalex.org/I201448701","display_name":"University of Washington","ror":"https://ror.org/00cvxb145","country_code":"US","type":"education","lineage":["https://openalex.org/I201448701"]},{"id":"https://openalex.org/I4405262941","display_name":"Seattle Children's Research Institute","ror":"https://ror.org/00cz0md82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1295499534","https://openalex.org/I4405262941"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Hans D. Ochs","raw_affiliation_strings":["Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","Department of Pediatrics, University of Washington, Seattle, WA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","institution_ids":["https://openalex.org/I4405262941"]},{"raw_affiliation_string":"Department of Pediatrics, University of Washington, Seattle, WA","institution_ids":["https://openalex.org/I201448701"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5027470095","display_name":"David J. Rawlings","orcid":"https://orcid.org/0000-0002-0810-1776","observed_orcids":["https://orcid.org/0000-0002-0810-1776","https://orcid.org/0000-0003-4370-4772"]},"institutions":[{"id":"https://openalex.org/I201448701","display_name":"University of Washington","ror":"https://ror.org/00cvxb145","country_code":"US","type":"education","lineage":["https://openalex.org/I201448701"]},{"id":"https://openalex.org/I4405262941","display_name":"Seattle Children's Research Institute","ror":"https://ror.org/00cz0md82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1295499534","https://openalex.org/I4405262941"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"David J. Rawlings","raw_affiliation_strings":["Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","Department of Pediatrics, University of Washington, Seattle, WA"],"raw_orcid":"https://orcid.org/0000-0002-0810-1776","affiliations":[{"raw_affiliation_string":"Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","institution_ids":["https://openalex.org/I4405262941"]},{"raw_affiliation_string":"Department of Pediatrics, University of Washington, Seattle, WA","institution_ids":["https://openalex.org/I201448701"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5022447244","display_name":"Carol H. Miao","orcid":"https://orcid.org/0000-0001-6520-2373","observed_orcids":["https://orcid.org/0000-0001-6520-2373"]},"institutions":[{"id":"https://openalex.org/I201448701","display_name":"University of Washington","ror":"https://ror.org/00cvxb145","country_code":"US","type":"education","lineage":["https://openalex.org/I201448701"]},{"id":"https://openalex.org/I4405262941","display_name":"Seattle Children's Research Institute","ror":"https://ror.org/00cz0md82","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1295499534","https://openalex.org/I4405262941"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Carol H. Miao","raw_affiliation_strings":["Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","Department of Pediatrics, University of Washington, Seattle, WA"],"raw_orcid":"https://orcid.org/0000-0001-6520-2373","affiliations":[{"raw_affiliation_string":"Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA","institution_ids":["https://openalex.org/I4405262941"]},{"raw_affiliation_string":"Department of Pediatrics, University of Washington, Seattle, WA","institution_ids":["https://openalex.org/I201448701"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":4,"corresponding_author_ids":["https://openalex.org/A5022447244"],"corresponding_institution_ids":["https://openalex.org/I201448701","https://openalex.org/I4405262941"],"apc_list":{"value":3000,"currency":"USD","value_usd":3000},"apc_paid":{"value":3000,"currency":"USD","value_usd":3000},"fwci":0.0,"has_fulltext":false,"cited_by_count":0,"citation_normalized_percentile":{"value":0.35144617,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":null,"biblio":{"volume":"3","issue":"1","first_page":"100119","last_page":"100119"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11280","display_name":"Hemophilia Treatment and Research","score":0.9160000085830688,"subfield":{"id":"https://openalex.org/subfields/2720","display_name":"Hematology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T11280","display_name":"Hemophilia Treatment and Research","score":0.9160000085830688,"subfield":{"id":"https://openalex.org/subfields/2720","display_name":"Hematology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.0828000009059906,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10746","display_name":"Platelet Disorders and Treatments","score":0.00019999999494757503,"subfield":{"id":"https://openalex.org/subfields/2720","display_name":"Hematology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/hemophilia-a","display_name":"hemophilia A","score":0.9710000157356262},{"id":"https://openalex.org/keywords/lentiviral-vectors","display_name":"lentiviral vectors","score":0.9589999914169312},{"id":"https://openalex.org/keywords/canine-model","display_name":"canine model","score":0.7490000128746033},{"id":"https://openalex.org/keywords/transgene-persistence","display_name":"transgene persistence","score":0.6589999794960022},{"id":"https://openalex.org/keywords/hemostasis","display_name":"hemostasis","score":0.8080000281333923},{"id":"https://openalex.org/keywords/bleeding-events","display_name":"bleeding events","score":0.8980000019073486},{"id":"https://openalex.org/keywords/transgene-copy-number","display_name":"transgene copy number","score":0.5490000247955322},{"id":"https://openalex.org/keywords/gene-therapy-safety","display_name":"gene therapy safety","score":0.4569999873638153}],"concepts":[{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.8213000297546387},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.7006000280380249},{"id":"https://openalex.org/C2778589496","wikidata":"https://www.wikidata.org/wiki/Q487937","display_name":"Hemostasis","level":2,"score":0.5935999751091003},{"id":"https://openalex.org/C89560881","wikidata":"https://www.wikidata.org/wiki/Q101026","display_name":"Platelet","level":2,"score":0.5404999852180481},{"id":"https://openalex.org/C2780007613","wikidata":"https://www.wikidata.org/wiki/Q546523","display_name":"Bone marrow","level":2,"score":0.5117999911308289},{"id":"https://openalex.org/C137061746","wikidata":"https://www.wikidata.org/wiki/Q736033","display_name":"Peripheral blood mononuclear cell","level":3,"score":0.49939998984336853},{"id":"https://openalex.org/C92087593","wikidata":"https://www.wikidata.org/wiki/Q427389","display_name":"Vector (molecular biology)","level":4,"score":0.4302000105381012},{"id":"https://openalex.org/C2781221834","wikidata":"https://www.wikidata.org/wiki/Q425360","display_name":"Factor IX","level":2,"score":0.41909998655319214},{"id":"https://openalex.org/C150194340","wikidata":"https://www.wikidata.org/wiki/Q26972","display_name":"Gene expression","level":3,"score":0.41679999232292175},{"id":"https://openalex.org/C135983454","wikidata":"https://www.wikidata.org/wiki/Q2790163","display_name":"Gene delivery","level":4,"score":0.414000004529953},{"id":"https://openalex.org/C207001950","wikidata":"https://www.wikidata.org/wiki/Q141124","display_name":"In vivo","level":2,"score":0.4077000021934509},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.3831999897956848},{"id":"https://openalex.org/C102230213","wikidata":"https://www.wikidata.org/wiki/Q430117","display_name":"Transgene","level":3,"score":0.3817000091075897},{"id":"https://openalex.org/C2777664302","wikidata":"https://www.wikidata.org/wiki/Q4463725","display_name":"Thromboelastography","level":3,"score":0.38029998540878296},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.38019999861717224},{"id":"https://openalex.org/C2992139802","wikidata":"https://www.wikidata.org/wiki/Q179217","display_name":"Clotting factor","level":2,"score":0.3776000142097473},{"id":"https://openalex.org/C2779216453","wikidata":"https://www.wikidata.org/wiki/Q489957","display_name":"Bleeding time","level":4,"score":0.37529999017715454},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.3668999969959259},{"id":"https://openalex.org/C141071460","wikidata":"https://www.wikidata.org/wiki/Q40821","display_name":"Surgery","level":1,"score":0.3479999899864197},{"id":"https://openalex.org/C194409129","wikidata":"https://www.wikidata.org/wiki/Q103824","display_name":"Hematology","level":2,"score":0.34049999713897705},{"id":"https://openalex.org/C98274493","wikidata":"https://www.wikidata.org/wiki/Q128406","display_name":"Pharmacology","level":1,"score":0.32760000228881836},{"id":"https://openalex.org/C2778382381","wikidata":"https://www.wikidata.org/wiki/Q179217","display_name":"Coagulation","level":2,"score":0.32739999890327454},{"id":"https://openalex.org/C43953973","wikidata":"https://www.wikidata.org/wiki/Q7917858","display_name":"Vectors in gene therapy","level":5,"score":0.3246000111103058},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.3077999949455261},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.289900004863739},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.2897999882698059},{"id":"https://openalex.org/C77171857","wikidata":"https://www.wikidata.org/wiki/Q5135593","display_name":"Clotting time","level":3,"score":0.2872999906539917},{"id":"https://openalex.org/C48023723","wikidata":"https://www.wikidata.org/wiki/Q856198","display_name":"Real-time polymerase chain reaction","level":3,"score":0.26919999718666077},{"id":"https://openalex.org/C2779936836","wikidata":"https://www.wikidata.org/wiki/Q890200","display_name":"Coagulopathy","level":2,"score":0.26080000400543213}],"mesh":[],"locations_count":6,"locations":[{"id":"doi:10.1016/j.bvth.2025.100119","doi":"https://doi.org/10.1016/j.bvth.2025.100119","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.bvth.2025.100119","pdf_url":null,"source":{"id":"https://openalex.org/S5407037026","display_name":"Blood Vessels Thrombosis & Hemostasis","issn_l":"2950-3272","issn":["2950-3272"],"is_oa":true,"is_in_doaj":true,"is_core":false,"listed_in":["doaj","ki-jl-1"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood Vessels, Thrombosis &amp; Hemostasis","raw_type":"journal-article"},{"id":"pmid:41608029","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/41608029","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood vessels, thrombosis & hemostasis","raw_type":"Journal Article"},{"id":"pmh:oai:pubmedcentral.nih.gov:12835592","doi":null,"is_oa":true,"landing_page_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC12835592/","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood Vessel Thromb Hemost","raw_type":"Text"},{"id":"pmh:cdr.lib.unc.edu:nc581352k","doi":null,"is_oa":true,"landing_page_url":"https://doi.org/10.17615/86wd-e331","pdf_url":null,"source":{"id":"https://openalex.org/S4306401075","display_name":"Carolina Digital Repository (University of North Carolina at Chapel Hill)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I114027177","host_organization_name":"University of North Carolina at Chapel Hill","host_organization_lineage":["https://openalex.org/I114027177"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Blood Vessels, Thrombosis & Hemostasis, 3(1)","raw_type":"Article"},{"id":"pmh:oai:doaj.org/article:e6c6c2da95804fe7b329b61143c00904","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/e6c6c2da95804fe7b329b61143c00904","pdf_url":null,"source":{"id":"https://openalex.org/S4306401280","display_name":"DOAJ (DOAJ: Directory of Open Access Journals)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-sa","license_id":"https://openalex.org/licenses/cc-by-sa","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Blood Vessels, Thrombosis & Hemostasis, Vol 3, Iss 1, Pp 100119- (2026)","raw_type":"article"},{"id":"doi:10.17615/86wd-e331","doi":"https://doi.org/10.17615/86wd-e331","is_oa":true,"landing_page_url":"https://doi.org/10.17615/86wd-e331","pdf_url":null,"source":{"id":"https://openalex.org/S7407051488","display_name":"UNC Libraries","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":null,"is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":"article-journal"}],"best_oa_location":{"id":"doi:10.1016/j.bvth.2025.100119","doi":"https://doi.org/10.1016/j.bvth.2025.100119","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.bvth.2025.100119","pdf_url":null,"source":{"id":"https://openalex.org/S5407037026","display_name":"Blood Vessels Thrombosis & Hemostasis","issn_l":"2950-3272","issn":["2950-3272"],"is_oa":true,"is_in_doaj":true,"is_core":false,"listed_in":["doaj","ki-jl-1"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood Vessels, Thrombosis &amp; Hemostasis","raw_type":"journal-article"},"sustainable_development_goals":[{"display_name":"Good health and well-being","score":0.9763000011444092,"id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G2835667028","display_name":null,"funder_award_id":"N01 75N92019D00041","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G2842358116","display_name":"Direct in vivo bone marrow transfer of lentiviral vector to correct hemophilia A","funder_award_id":"R56HL123326","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G4873398043","display_name":"Direct in vivo bone marrow transfer of lentiviral vector to correct hemophilia A","funder_award_id":"R01HL123326","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G575533958","display_name":"Ultrasound-mediated gene delivery to achieve therapeutic correction of hemophilia A","funder_award_id":"R01HL151077","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G8776146842","display_name":"Intraosseous delivery of lentiviral vectors for hemophilia A gene therapy","funder_award_id":"R01HL134321","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G9000879739","display_name":"Gene Editing for Hemophilia A Treatment Using Lipid Nanoparticles","funder_award_id":"R01HL169793","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G9000914114","display_name":"New Mechanistic Insights & Therapeutic Applications of Megakaryocytes/Platelets","funder_award_id":"R35HL150698","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"}],"funders":[{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"},{"id":"https://openalex.org/F4320337338","display_name":"National Heart, Lung, and Blood Institute","ror":"https://ror.org/012pb6c26"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":40,"referenced_works":["https://openalex.org/W230816652","https://openalex.org/W1543154092","https://openalex.org/W1890557990","https://openalex.org/W1977493474","https://openalex.org/W1983197092","https://openalex.org/W1994064996","https://openalex.org/W1994849771","https://openalex.org/W2008004245","https://openalex.org/W2016608925","https://openalex.org/W2041489036","https://openalex.org/W2043626618","https://openalex.org/W2045245489","https://openalex.org/W2052207235","https://openalex.org/W2058197610","https://openalex.org/W2059303794","https://openalex.org/W2061584786","https://openalex.org/W2069848235","https://openalex.org/W2082337683","https://openalex.org/W2096674194","https://openalex.org/W2170341515","https://openalex.org/W2170563066","https://openalex.org/W2277190971","https://openalex.org/W2284642874","https://openalex.org/W2336872964","https://openalex.org/W2518229080","https://openalex.org/W2527986046","https://openalex.org/W2611005000","https://openalex.org/W2782917400","https://openalex.org/W2788609897","https://openalex.org/W3017135170","https://openalex.org/W3021433538","https://openalex.org/W3087211738","https://openalex.org/W3181927643","https://openalex.org/W3185795374","https://openalex.org/W4285726658","https://openalex.org/W4293708402","https://openalex.org/W4321482598","https://openalex.org/W4405175875","https://openalex.org/W4406722797","https://openalex.org/W4408893301"],"related_works":["https://openalex.org/W2414609756","https://openalex.org/W2039928766","https://openalex.org/W2582219035","https://openalex.org/W1975525138","https://openalex.org/W2527986046","https://openalex.org/W2579040422","https://openalex.org/W2562587463","https://openalex.org/W2059303794","https://openalex.org/W2102016263","https://openalex.org/W7096382823"],"abstract_inverted_index":{"1.":[0],"Hemophilia":[1,37],"A":[2,35,38],"dogs":[3,107,137,240],"treated":[4,33,87,176,234,239],"with":[5,158,268],"intraosseous":[6,68],"infusions":[7,53],"of":[8,74,94,105,132,180],"platelet-specific":[9,117],"FVIII-LVs":[10],"achieved":[11],"partial":[12],"phenotypic":[13],"correction.":[14],"2.":[15],"Canine":[16,151],"FVIII":[17,82,152],"could":[18],"be":[19,154],"detected":[20,155,185],"in":[21,32,84,156,174,209,233,260],"platelets":[22,85,157],"over":[23],"2":[24],"years":[25],"and":[26,60,120,143,166,202,227,258],"bleeding":[27,243],"events":[28,244],"were":[29,183],"significantly":[30],"reduced":[31],"hemophilia":[34],"dogs.":[36,135,177,235],"(HemA)":[39],"is":[40],"a":[41,46,116,255],"genetic":[42],"disease":[43],"resulting":[44],"from":[45,140,186,191],"factor":[47],"VIII":[48],"(FVIII)":[49],"deficiency.":[50],"Traditional":[51],"protein":[52],"to":[54,98],"treat":[55],"HemA":[56,88,106,134],"are":[57],"costly,":[58],"inconvenient,":[59],"requires":[61],"repeated":[62],"dosing.":[63],"We":[64],"demonstrated":[65,254],"previously":[66],"that":[67],"(IO)":[69],"gene":[70,110,123,217,222,248,262],"therapy":[71,223,249,263],"via":[72],"delivery":[73],"lentiviral":[75],"vectors":[76],"(LVs)":[77],"into":[78,126],"bone":[79],"marrow":[80],"targeting":[81],"expression":[83,161,168],"successfully":[86],"mice.":[89],"To":[90],"facilitate":[91],"the":[92,103,127,141,159,167,171,181,187,220,238],"translation":[93],"this":[95],"novel":[96],"strategy":[97,264],"clinical":[99],"application,":[100],"we":[101],"investigated":[102],"treatment":[104],"using":[108],"IO":[109,221],"therapy.":[111,218],"The":[112,196],"VSVG-pseudotyped":[113],"G-cF8-LV":[114],"incorporating":[115],"promoter":[118],"Gp1b\u03b1":[119],"canine":[121],"F8":[122],"was":[124,224],"injected":[125],"tibia":[128],"or":[129],"iliac":[130],"bones":[131],"4":[133],"All":[136],"recovered":[138],"well":[139,225],"procedure":[142],"had":[144],"blood":[145,194,199],"chemistry":[146],"values":[147],"within":[148],"normal":[149],"ranges.":[150],"can":[153],"highest":[160],"around":[162],"40-50":[163],"days":[164],"post-procedure":[165],"persisted":[169],"for":[170,265],"experimental":[172],"duration":[173],"all":[175],"Copy":[178],"numbers":[179],"transgene":[182],"persistently":[184],"genomic":[188],"DNA":[189],"isolated":[190],"peripheral":[192],"mononuclear":[193],"cells.":[195],"shortened":[197],"whole":[198],"clotting":[200],"time":[201,211],"improved":[203,214],"parameters":[204],"evaluated":[205],"by":[206],"thromboelastography":[207],"testing":[208],"multiple":[210],"points":[212],"indicated":[213],"hemostasis":[215],"after":[216,247],"Furthermore,":[219],"tolerated":[226],"did":[228],"not":[229],"produce":[230],"any":[231],"toxicity":[232],"Most":[236],"significantly,":[237],"experienced":[241],"fewer":[242],"per":[245],"year":[246],"than":[250],"prior.":[251],"Our":[252],"study":[253],"potentially":[256],"safe":[257],"effective":[259],"vivo":[261],"treating":[266],"people":[267],"HemA.":[269]},"counts_by_year":[],"updated_date":"2026-10-30T12:39:25.321706","created_date":"2025-10-25T00:00:00"}
