{"id":"https://openalex.org/W3011016243","doi":"https://doi.org/10.1002/sctm.19-0261","title":"Endothelial cells derived from patients\u2019 induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A","display_name":"Endothelial cells derived from patients\u2019 induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A","publication_year":2020,"publication_date":"2020-03-12","ids":{"openalex":"https://openalex.org/W3011016243","doi":"https://doi.org/10.1002/sctm.19-0261","mag":"3011016243","pmid":"https://pubmed.ncbi.nlm.nih.gov/32162786","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/7214661"},"language":"en","primary_location":{"id":"doi:10.1002/sctm.19-0261","doi":"https://doi.org/10.1002/sctm.19-0261","is_oa":true,"landing_page_url":"https://doi.org/10.1002/sctm.19-0261","pdf_url":"https://stemcellsjournals.onlinelibrary.wiley.com/doi/pdfdirect/10.1002/sctm.19-0261","source":{"id":"https://openalex.org/S2494728167","display_name":"Stem Cells Translational Medicine","issn_l":"2157-6564","issn":["2157-6564","2157-6580"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-a","doyens","jufo-2","ki-jl-1","medline","norway-1","oarl","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Stem Cells Translational Medicine","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://stemcellsjournals.onlinelibrary.wiley.com/doi/pdfdirect/10.1002/sctm.19-0261","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5021842003","display_name":"Melanie D. Rose","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160527","display_name":"University of California Davis Medical Center","ror":"https://ror.org/05t6gpm70","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160527","https://openalex.org/I4210160856"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Melanie Rose","raw_affiliation_strings":["1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California","institution_ids":["https://openalex.org/I4210160527"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5049587502","display_name":"Kewa Gao","orcid":"https://orcid.org/0000-0002-8781-890X","observed_orcids":["https://orcid.org/0000-0002-8781-890X"]},"institutions":[{"id":"https://openalex.org/I4210103356","display_name":"Shriners Hospitals for Children - Northern California","ror":"https://ror.org/01dmyj570","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210103356","https://openalex.org/I4210144267"]},{"id":"https://openalex.org/I4210144267","display_name":"Shriners Hospitals for Children","ror":"https://ror.org/03e8tm275","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210144267"]},{"id":"https://openalex.org/I4210160527","display_name":"University of California Davis Medical Center","ror":"https://ror.org/05t6gpm70","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160527","https://openalex.org/I4210160856"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Kewa Gao","raw_affiliation_strings":["2 Department of Surgery University of California Davis Medical Center, Sacramento, California","3 Institute for Pediatric Regenerative Medicine Shriners Hospitals for Children, Sacramento, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"2 Department of Surgery University of California Davis Medical Center, Sacramento, California","institution_ids":["https://openalex.org/I4210160527"]},{"raw_affiliation_string":"3 Institute for Pediatric Regenerative Medicine Shriners Hospitals for Children, Sacramento, California","institution_ids":["https://openalex.org/I4210103356","https://openalex.org/I4210144267"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5070456243","display_name":"Elizabeth Cortez-Toledo","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160527","display_name":"University of California Davis Medical Center","ror":"https://ror.org/05t6gpm70","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160527","https://openalex.org/I4210160856"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Elizabeth Cortez-Toledo","raw_affiliation_strings":["1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California","institution_ids":["https://openalex.org/I4210160527"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5072206841","display_name":"Emmanuel Agu","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160527","display_name":"University of California Davis Medical Center","ror":"https://ror.org/05t6gpm70","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160527","https://openalex.org/I4210160856"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Emmanuel Agu","raw_affiliation_strings":["1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California","institution_ids":["https://openalex.org/I4210160527"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5001297620","display_name":"Alicia A. Hyllen","orcid":"https://orcid.org/0000-0002-9317-9366","observed_orcids":["https://orcid.org/0000-0002-9317-9366"]},"institutions":[{"id":"https://openalex.org/I4210160527","display_name":"University of California Davis Medical Center","ror":"https://ror.org/05t6gpm70","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160527","https://openalex.org/I4210160856"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Alicia A. Hyllen","raw_affiliation_strings":["1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California","institution_ids":["https://openalex.org/I4210160527"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5003435352","display_name":"Kelsey L Conroy","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160527","display_name":"University of California Davis Medical Center","ror":"https://ror.org/05t6gpm70","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160527","https://openalex.org/I4210160856"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Kelsey Conroy","raw_affiliation_strings":["1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California","institution_ids":["https://openalex.org/I4210160527"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5037315167","display_name":"Guangjin Pan","orcid":"https://orcid.org/0000-0002-2378-7198","observed_orcids":["https://orcid.org/0000-0002-2378-7198"]},"institutions":[{"id":"https://openalex.org/I19820366","display_name":"Chinese Academy of Sciences","ror":"https://ror.org/034t30j35","country_code":"CN","type":"government","lineage":["https://openalex.org/I19820366"]},{"id":"https://openalex.org/I2802148028","display_name":"Guangzhou Institutes of Biomedicine and Health","ror":"https://ror.org/02c31t502","country_code":"CN","type":"facility","lineage":["https://openalex.org/I19820366","https://openalex.org/I2802148028"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Guangjin Pan","raw_affiliation_strings":["4 Key Laboratory of Regenerative Biology Guangzhou Institute of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, China"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"4 Key Laboratory of Regenerative Biology Guangzhou Institute of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, China","institution_ids":["https://openalex.org/I19820366","https://openalex.org/I2802148028"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5085630765","display_name":"Jan A. Nolta","orcid":"https://orcid.org/0000-0003-4576-8542","observed_orcids":["https://orcid.org/0000-0003-4576-8542"]},"institutions":[{"id":"https://openalex.org/I4210160527","display_name":"University of California Davis Medical Center","ror":"https://ror.org/05t6gpm70","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160527","https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jan A. Nolta","raw_affiliation_strings":["1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California","5 University of California Davis Gene Therapy Center, Sacramento, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California","institution_ids":["https://openalex.org/I4210160527"]},{"raw_affiliation_string":"5 University of California Davis Gene Therapy Center, Sacramento, California","institution_ids":["https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5100675356","display_name":"Aijun Wang","orcid":"https://orcid.org/0000-0002-2985-3627","observed_orcids":["https://orcid.org/0000-0002-2985-3627"]},"institutions":[{"id":"https://openalex.org/I4210103356","display_name":"Shriners Hospitals for Children - Northern California","ror":"https://ror.org/01dmyj570","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210103356","https://openalex.org/I4210144267"]},{"id":"https://openalex.org/I4210144267","display_name":"Shriners Hospitals for Children","ror":"https://ror.org/03e8tm275","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210144267"]},{"id":"https://openalex.org/I4210160527","display_name":"University of California Davis Medical Center","ror":"https://ror.org/05t6gpm70","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160527","https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Aijun Wang","raw_affiliation_strings":["2 Department of Surgery University of California Davis Medical Center, Sacramento, California","3 Institute for Pediatric Regenerative Medicine Shriners Hospitals for Children, Sacramento, California","6 Department of Biomedical Engineering University of California Davis, Davis, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"2 Department of Surgery University of California Davis Medical Center, Sacramento, California","institution_ids":["https://openalex.org/I4210160527"]},{"raw_affiliation_string":"3 Institute for Pediatric Regenerative Medicine Shriners Hospitals for Children, Sacramento, California","institution_ids":["https://openalex.org/I4210103356","https://openalex.org/I4210144267"]},{"raw_affiliation_string":"6 Department of Biomedical Engineering University of California Davis, Davis, California","institution_ids":["https://openalex.org/I84218800"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5103049750","display_name":"Ping Zhou","orcid":"https://orcid.org/0000-0002-6644-788X","observed_orcids":["https://orcid.org/0000-0002-6644-788X"]},"institutions":[{"id":"https://openalex.org/I4210160527","display_name":"University of California Davis Medical Center","ror":"https://ror.org/05t6gpm70","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160527","https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Ping Zhou","raw_affiliation_strings":["1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California","5 University of California Davis Gene Therapy Center, Sacramento, California"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"1 Stem Cell Program, Department of Internal Medicine University of California Davis Medical Center, Sacramento, California","institution_ids":["https://openalex.org/I4210160527"]},{"raw_affiliation_string":"5 University of California Davis Gene Therapy Center, Sacramento, California","institution_ids":["https://openalex.org/I84218800"]}]}],"institutions":[],"countries_distinct_count":2,"institutions_distinct_count":6,"corresponding_author_ids":["https://openalex.org/A5100675356","https://openalex.org/A5103049750"],"corresponding_institution_ids":["https://openalex.org/I4210103356","https://openalex.org/I4210144267","https://openalex.org/I4210160527","https://openalex.org/I84218800"],"apc_list":{"value":2200,"currency":"USD","value_usd":2200},"apc_paid":{"value":2200,"currency":"USD","value_usd":2200},"fwci":2.6833,"has_fulltext":true,"cited_by_count":22,"citation_normalized_percentile":{"value":0.91008583,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":94,"max":98},"biblio":{"volume":"9","issue":"6","first_page":"686","last_page":"696"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11280","display_name":"Hemophilia Treatment and Research","score":0.9970999956130981,"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":0.9970999956130981,"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":0.0017000000225380063,"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/T10505","display_name":"Pluripotent Stem Cells Research","score":0.0003000000142492354,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"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"}}],"keywords":[{"id":"https://openalex.org/keywords/hemophilia-a","display_name":"hemophilia A","score":1.0},{"id":"https://openalex.org/keywords/endothelial-cells","display_name":"endothelial cells","score":0.7749999761581421},{"id":"https://openalex.org/keywords/induced-pluripotent-stem-cells","display_name":"induced pluripotent stem cells","score":0.9879999756813049},{"id":"https://openalex.org/keywords/cell-therapy","display_name":"cell therapy","score":0.7200000286102295},{"id":"https://openalex.org/keywords/lentiviral-vectors","display_name":"lentiviral vectors","score":0.7570000290870667},{"id":"https://openalex.org/keywords/b-domain-deleted-factor-viii","display_name":"B-domain-deleted factor VIII","score":0.7129999995231628},{"id":"https://openalex.org/keywords/autologous-cell-therapy","display_name":"autologous cell therapy","score":0.8029999732971191},{"id":"https://openalex.org/keywords/engraftment","display_name":"engraftment","score":0.7490000128746033},{"id":"https://openalex.org/keywords/bioluminescence-imaging","display_name":"bioluminescence imaging","score":0.7279999852180481}],"concepts":[{"id":"https://openalex.org/C107459253","wikidata":"https://www.wikidata.org/wiki/Q674925","display_name":"Induced pluripotent stem cell","level":4,"score":0.8645042777061462},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.6249151825904846},{"id":"https://openalex.org/C28328180","wikidata":"https://www.wikidata.org/wiki/Q48196","display_name":"Stem cell","level":2,"score":0.515896201133728},{"id":"https://openalex.org/C123012128","wikidata":"https://www.wikidata.org/wiki/Q5376430","display_name":"Endothelial stem cell","level":3,"score":0.4907778203487396},{"id":"https://openalex.org/C2778939058","wikidata":"https://www.wikidata.org/wiki/Q4046167","display_name":"CD31","level":3,"score":0.4636249542236328},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.458187460899353},{"id":"https://openalex.org/C161997846","wikidata":"https://www.wikidata.org/wiki/Q4915066","display_name":"Bioluminescence imaging","level":5,"score":0.45311176776885986},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.43318843841552734},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.42337167263031006},{"id":"https://openalex.org/C502942594","wikidata":"https://www.wikidata.org/wiki/Q3421914","display_name":"Cancer research","level":1,"score":0.38434016704559326},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.366074800491333},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.3523637056350708},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.31310755014419556},{"id":"https://openalex.org/C54009773","wikidata":"https://www.wikidata.org/wiki/Q1429031","display_name":"Transfection","level":3,"score":0.3115982711315155},{"id":"https://openalex.org/C145103041","wikidata":"https://www.wikidata.org/wiki/Q1151519","display_name":"Embryonic stem cell","level":3,"score":0.28476399183273315},{"id":"https://openalex.org/C111335760","wikidata":"https://www.wikidata.org/wiki/Q95385166","display_name":"Luciferase","level":4,"score":0.28121453523635864},{"id":"https://openalex.org/C81885089","wikidata":"https://www.wikidata.org/wiki/Q189082","display_name":"Cell culture","level":2,"score":0.201593279838562},{"id":"https://openalex.org/C2780394083","wikidata":"https://www.wikidata.org/wiki/Q539568","display_name":"Angiogenesis","level":2,"score":0.1979643702507019},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.17004474997520447},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.12164518237113953},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.10612910985946655},{"id":"https://openalex.org/C202751555","wikidata":"https://www.wikidata.org/wiki/Q221681","display_name":"In vitro","level":2,"score":0.07673156261444092}],"mesh":[{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002454","descriptor_name":"Cell Differentiation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004195","descriptor_name":"Disease Models, Animal","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005169","descriptor_name":"Factor VIII","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D005169","descriptor_name":"Factor VIII","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008810","descriptor_name":"Mice, Inbred C57BL","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D042783","descriptor_name":"Endothelial Cells","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":true},{"descriptor_ui":"D042783","descriptor_name":"Endothelial Cells","qualifier_ui":"Q000637","qualifier_name":"transplantation","is_major_topic":false},{"descriptor_ui":"D049452","descriptor_name":"Green Fluorescent Proteins","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D057026","descriptor_name":"Induced Pluripotent Stem Cells","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":true},{"descriptor_ui":"D057026","descriptor_name":"Induced Pluripotent Stem Cells","qualifier_ui":"Q000637","qualifier_name":"transplantation","is_major_topic":false}],"locations_count":6,"locations":[{"id":"doi:10.1002/sctm.19-0261","doi":"https://doi.org/10.1002/sctm.19-0261","is_oa":true,"landing_page_url":"https://doi.org/10.1002/sctm.19-0261","pdf_url":"https://stemcellsjournals.onlinelibrary.wiley.com/doi/pdfdirect/10.1002/sctm.19-0261","source":{"id":"https://openalex.org/S2494728167","display_name":"Stem Cells Translational Medicine","issn_l":"2157-6564","issn":["2157-6564","2157-6580"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-a","doyens","jufo-2","ki-jl-1","medline","norway-1","oarl","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Stem Cells Translational Medicine","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:7214661","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/7214661","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","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Stem Cells Transl Med","raw_type":"Text"},{"id":"pmid:32162786","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/32162786","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":"Stem cells translational medicine","raw_type":"Journal Article"},{"id":"pmh:oai:doaj.org/article:9b49c5c3207b4640b4388e1e638ad24a","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/9b49c5c3207b4640b4388e1e638ad24a","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":"Stem Cells Translational Medicine, Vol 9, Iss 6, Pp 686-696 (2020)","raw_type":"article"},{"id":"pmh:oai:escholarship.org/ark:/13030/qt45p275gt","doi":null,"is_oa":false,"landing_page_url":"https://escholarship.org/uc/item/45p275gt","pdf_url":null,"source":{"id":"https://openalex.org/S4306400115","display_name":"eScholarship (California Digital Library)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2801248553","host_organization_name":"California Digital Library","host_organization_lineage":["https://openalex.org/I2801248553"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Stem cells translational medicine, vol 9, iss 6","raw_type":"article"},{"id":"pmh:oai:escholarship.org:ark:/13030/qt45p275gt","doi":null,"is_oa":true,"landing_page_url":"https://escholarship.org/uc/item/45p275gt","pdf_url":null,"source":{"id":"https://openalex.org/S4306400115","display_name":"eScholarship (California Digital Library)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2801248553","host_organization_name":"California Digital Library","host_organization_lineage":["https://openalex.org/I2801248553"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Stem Cells Translational Medicine, vol 9, iss 6","raw_type":"article"}],"best_oa_location":{"id":"doi:10.1002/sctm.19-0261","doi":"https://doi.org/10.1002/sctm.19-0261","is_oa":true,"landing_page_url":"https://doi.org/10.1002/sctm.19-0261","pdf_url":"https://stemcellsjournals.onlinelibrary.wiley.com/doi/pdfdirect/10.1002/sctm.19-0261","source":{"id":"https://openalex.org/S2494728167","display_name":"Stem Cells Translational Medicine","issn_l":"2157-6564","issn":["2157-6564","2157-6580"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-a","doyens","jufo-2","ki-jl-1","medline","norway-1","oarl","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Stem Cells Translational Medicine","raw_type":"journal-article"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being","score":1.0}],"study_designs":[],"awards":[{"id":"https://openalex.org/G3608626411","display_name":null,"funder_award_id":"84705-NCA-19","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G3641269095","display_name":"Direct cell to cell transfer of microRNA for tissue repair","funder_award_id":"R01GM099688","funder_id":"https://openalex.org/F4320337354","funder_display_name":"National Institute of General Medical Sciences"},{"id":"https://openalex.org/G5520316217","display_name":null,"funder_award_id":"NIH-R01","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G7158873286","display_name":null,"funder_award_id":"84705\u2010NCA\u201019","funder_id":"https://openalex.org/F4320313002","funder_display_name":"Shriners Hospitals for Children"},{"id":"https://openalex.org/G8112734794","display_name":"Strengthening the Pipeline of Masters-level Scientific and Laboratory Personnel in Stem Cell Research","funder_award_id":"TB1-01184","funder_id":"https://openalex.org/F4320306171","funder_display_name":"California Institute for Regenerative Medicine"},{"id":"https://openalex.org/G8779155704","display_name":null,"funder_award_id":"84705-NCA-19","funder_id":"https://openalex.org/F4320306171","funder_display_name":"California Institute for Regenerative Medicine"}],"funders":[{"id":"https://openalex.org/F4320306171","display_name":"California Institute for Regenerative Medicine","ror":"https://ror.org/033m8b439"},{"id":"https://openalex.org/F4320310280","display_name":"California State University","ror":"https://ror.org/020qm1538"},{"id":"https://openalex.org/F4320313002","display_name":"Shriners Hospitals for Children","ror":"https://ror.org/03e8tm275"},{"id":"https://openalex.org/F4320314994","display_name":"Milstein Medical Asian American Partnership Foundation","ror":"https://ror.org/01readg34"},{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"},{"id":"https://openalex.org/F4320332564","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69"},{"id":"https://openalex.org/F4320337354","display_name":"National Institute of General Medical Sciences","ror":"https://ror.org/04q48ey07"},{"id":"https://openalex.org/F4320337873","display_name":"University of California Davis School of Medicine","ror":null}],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W3011016243.pdf","grobid_xml":"https://content.openalex.org/works/W3011016243.grobid-xml"},"referenced_works_count":54,"referenced_works":["https://openalex.org/W1492446984","https://openalex.org/W1510414151","https://openalex.org/W1563180177","https://openalex.org/W1914919813","https://openalex.org/W1966225115","https://openalex.org/W1969659837","https://openalex.org/W1970302216","https://openalex.org/W1973690819","https://openalex.org/W1974736062","https://openalex.org/W1976329054","https://openalex.org/W1977471852","https://openalex.org/W1978668313","https://openalex.org/W1987059879","https://openalex.org/W2000194482","https://openalex.org/W2007261697","https://openalex.org/W2018509153","https://openalex.org/W2019893582","https://openalex.org/W2025242318","https://openalex.org/W2029922157","https://openalex.org/W2039928766","https://openalex.org/W2043167315","https://openalex.org/W2056162867","https://openalex.org/W2058468253","https://openalex.org/W2068859710","https://openalex.org/W2072802196","https://openalex.org/W2075623608","https://openalex.org/W2080943222","https://openalex.org/W2082929405","https://openalex.org/W2089865394","https://openalex.org/W2096844650","https://openalex.org/W2098669942","https://openalex.org/W2099598492","https://openalex.org/W2117060790","https://openalex.org/W2132330583","https://openalex.org/W2141301124","https://openalex.org/W2142046859","https://openalex.org/W2150001402","https://openalex.org/W2222135202","https://openalex.org/W2276582211","https://openalex.org/W2511036853","https://openalex.org/W2520151927","https://openalex.org/W2593871081","https://openalex.org/W2606688282","https://openalex.org/W2618806491","https://openalex.org/W2775538622","https://openalex.org/W2792153930","https://openalex.org/W2806820493","https://openalex.org/W2807198924","https://openalex.org/W2882112986","https://openalex.org/W2883697525","https://openalex.org/W2900205648","https://openalex.org/W2916215586","https://openalex.org/W2945199469","https://openalex.org/W2948541070"],"related_works":["https://openalex.org/W2900205648","https://openalex.org/W2566213919","https://openalex.org/W2752710363","https://openalex.org/W7070689700","https://openalex.org/W2502778056","https://openalex.org/W4213047173","https://openalex.org/W1974736062","https://openalex.org/W3207598312","https://openalex.org/W2994668604","https://openalex.org/W4406085550"],"abstract_inverted_index":{"Abstract":[0],"Hemophilia":[1],"A":[2,218],"(HA)":[3,219],"is":[4,16,35,290],"a":[5,43,54,71,162,185,291],"bleeding":[6,164],"disorder":[7],"characterized":[8],"by":[9,18,142,263],"spontaneous":[10],"and":[11,97,110,128,135,145,166,252,255],"prolonged":[12],"hemorrhage.":[13],"The":[14,76,205,230,242,287],"disease":[15],"caused":[17],"mutations":[19],"in":[20,38,42,120,161,197,201,249,266,271,295],"the":[21,87,94,98,115,121,136,191,256,272,275],"coagulation":[22],"factor":[23,29,240],"8":[24],"gene":[25,103],"(F8)":[26],"leading":[27],"to":[28,84,187,237],"VIII":[30],"(FVIII)":[31],"deficiency.":[32],"Since":[33],"FVIII":[34,189],"primarily":[36],"produced":[37,167],"endothelial":[39,137,227],"cells":[40,67,213,228],"(ECs)":[41],"non-diseased":[44],"human":[45],"being,":[46],"ECs":[47,78,117,156,181,232,265,283],"hold":[48],"great":[49],"potential":[50,278],"for":[51,57,123,190,277,284,302],"development":[52,280,296],"as":[53,140,184,269],"cell":[55,300],"therapy":[56,301],"HA.":[58,286,303],"We":[59],"showed":[60],"that":[61,179,209],"HA":[62,151,194],"patient-specific":[63],"induced":[64,210],"pluripotent":[65,211],"stem":[66,212],"(HA-iPSCs)":[68],"could":[69],"provide":[70,177,222,274],"renewable":[72],"supply":[73,225],"of":[74,132,172,193,226,246,261,281,297],"ECs.":[75],"HA-iPSC-derived":[77,116,155,180,231,282],"were":[79,118],"transduced":[80,154],"with":[81],"lentiviral":[82],"vectors":[83],"stably":[85],"express":[86],"functional":[88,239,257],"B":[89],"domain":[90],"deleted":[91],"F8":[92],"gene,":[93,96],"luciferase":[95],"enhanced":[99],"green":[100],"fluorescent":[101],"protein":[102],"(GFP).":[104],"When":[105,148],"transplanted":[106,149],"intramuscularly":[107],"into":[108,150],"neonatal":[109,251],"adult":[111,253],"immune":[112],"deficient":[113],"mice,":[114,152],"retained":[119],"animals":[122],"at":[124],"least":[125],"10-16":[126],"weeks":[127],"maintained":[129],"their":[130],"expression":[131],"FVIII,":[133],"GFP,":[134],"marker":[138],"CD31,":[139],"demonstrated":[141,208],"bioluminescence":[143],"imaging":[144],"immunostaining,":[146],"respectively.":[147],"these":[153,247,264],"significantly":[157],"reduced":[158],"blood":[159],"loss":[160],"tail-clip":[163],"test":[165],"therapeutic":[168,279],"plasma":[169],"levels":[170],"(11.2%-369.2%)":[171],"FVIII.":[173],"Thus,":[174],"our":[175],"studies":[176],"proof-of-concept":[178],"can":[182,221,233],"serve":[183],"factory":[186],"deliver":[188],"treatment":[192],"not":[195],"only":[196],"adults":[198],"but":[199],"also":[200],"newborns.":[202],"Significance":[203],"statement":[204],"present":[206],"study":[207,289],"(iPSCs)":[214],"derived":[215],"from":[216],"hemophilia":[217,262],"patients":[220],"an":[223],"ample":[224],"(ECs).":[229],"be":[234],"genetically":[235],"modified":[236],"produce":[238],"VIII.":[241],"relatively":[243],"stable":[244],"engraftment":[245],"ECs,":[248],"both":[250],"animals,":[254],"correction":[258],"or":[259],"alleviation":[260],"animal":[267],"models,":[268],"shown":[270],"study,":[273],"basis":[276],"treating":[285],"current":[288],"significant":[292],"step":[293],"forward":[294],"autologous":[298],"gene-modified":[299]},"counts_by_year":[{"year":2025,"cited_by_count":3},{"year":2024,"cited_by_count":2},{"year":2023,"cited_by_count":5},{"year":2022,"cited_by_count":4},{"year":2021,"cited_by_count":4},{"year":2020,"cited_by_count":4}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2020-03-23T00:00:00"}
