{"id":"https://openalex.org/W2018046746","doi":"https://doi.org/10.1182/blood-2004-03-1028","title":"Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene","display_name":"Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene","publication_year":2004,"publication_date":"2004-06-25","ids":{"openalex":"https://openalex.org/W2018046746","doi":"https://doi.org/10.1182/blood-2004-03-1028","mag":"2018046746","pmid":"https://pubmed.ncbi.nlm.nih.gov/15217833"},"language":"en","primary_location":{"id":"doi:10.1182/blood-2004-03-1028","doi":"https://doi.org/10.1182/blood-2004-03-1028","is_oa":false,"landing_page_url":"https://doi.org/10.1182/blood-2004-03-1028","pdf_url":null,"source":{"id":"https://openalex.org/S200071133","display_name":"Blood","issn_l":"0006-4971","issn":["0006-4971","1528-0020"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"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":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":false,"oa_status":"closed","oa_url":null,"any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5007360666","display_name":"Denise E. Sabatino","orcid":"https://orcid.org/0000-0002-6116-5453","observed_orcids":["https://orcid.org/0000-0002-6116-5453"]},"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"]},{"id":"https://openalex.org/I1344073410","display_name":"Howard Hughes Medical Institute","ror":"https://ror.org/006w34k90","country_code":"US","type":"facility","lineage":["https://openalex.org/I1344073410"]},{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Denise E. Sabatino","raw_affiliation_strings":["From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I1344073410","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5110071717","display_name":"Elina Armstrong","orcid":null,"observed_orcids":[]},"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"]},{"id":"https://openalex.org/I1344073410","display_name":"Howard Hughes Medical Institute","ror":"https://ror.org/006w34k90","country_code":"US","type":"facility","lineage":["https://openalex.org/I1344073410"]},{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Elina Armstrong","raw_affiliation_strings":["From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I1344073410","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5110240079","display_name":"Shyrie A. Edmonson","orcid":null,"observed_orcids":[]},"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"]},{"id":"https://openalex.org/I1344073410","display_name":"Howard Hughes Medical Institute","ror":"https://ror.org/006w34k90","country_code":"US","type":"facility","lineage":["https://openalex.org/I1344073410"]},{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Shyrie Edmonson","raw_affiliation_strings":["From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I1344073410","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Yi-Lin Liu","orcid":null,"observed_orcids":[]},"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"]},{"id":"https://openalex.org/I1344073410","display_name":"Howard Hughes Medical Institute","ror":"https://ror.org/006w34k90","country_code":"US","type":"facility","lineage":["https://openalex.org/I1344073410"]},{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Yi-Lin Liu","raw_affiliation_strings":["From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I1344073410","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5023101456","display_name":"Marc Pleimes","orcid":null,"observed_orcids":[]},"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"]},{"id":"https://openalex.org/I1344073410","display_name":"Howard Hughes Medical Institute","ror":"https://ror.org/006w34k90","country_code":"US","type":"facility","lineage":["https://openalex.org/I1344073410"]},{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Marc Pleimes","raw_affiliation_strings":["From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I1344073410","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5111970085","display_name":"J\u00f6rg Sch\u00fcttrumpf","orcid":"https://orcid.org/0000-0002-8855-4096","observed_orcids":["https://orcid.org/0000-0002-8855-4096"]},"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"]},{"id":"https://openalex.org/I1344073410","display_name":"Howard Hughes Medical Institute","ror":"https://ror.org/006w34k90","country_code":"US","type":"facility","lineage":["https://openalex.org/I1344073410"]},{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Joerg Schuettrumpf","raw_affiliation_strings":["From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I1344073410","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5088663182","display_name":"Julie C. Fitzgerald","orcid":"https://orcid.org/0000-0001-9936-9569","observed_orcids":["https://orcid.org/0000-0001-9936-9569"]},"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"]},{"id":"https://openalex.org/I1344073410","display_name":"Howard Hughes Medical Institute","ror":"https://ror.org/006w34k90","country_code":"US","type":"facility","lineage":["https://openalex.org/I1344073410"]},{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Julie Fitzgerald","raw_affiliation_strings":["From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I1344073410","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5086755266","display_name":"Roland W. Herzog","orcid":"https://orcid.org/0000-0002-7213-998X","observed_orcids":["https://orcid.org/0000-0002-7213-998X","https://orcid.org/0000-0003-2164-6575"]},"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"]},{"id":"https://openalex.org/I1344073410","display_name":"Howard Hughes Medical Institute","ror":"https://ror.org/006w34k90","country_code":"US","type":"facility","lineage":["https://openalex.org/I1344073410"]},{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Roland W. Herzog","raw_affiliation_strings":["From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I1344073410","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5083658192","display_name":"Valder R. Arruda","orcid":"https://orcid.org/0000-0002-8100-5481","observed_orcids":["https://orcid.org/0000-0002-8100-5481"]},"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"]},{"id":"https://openalex.org/I1344073410","display_name":"Howard Hughes Medical Institute","ror":"https://ror.org/006w34k90","country_code":"US","type":"facility","lineage":["https://openalex.org/I1344073410"]},{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Valder R. Arruda","raw_affiliation_strings":["From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I1344073410","https://openalex.org/I79576946"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5060779925","display_name":"Katherine A. High","orcid":"https://orcid.org/0000-0002-0805-6223","observed_orcids":["https://orcid.org/0000-0002-0805-6223"]},"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"]},{"id":"https://openalex.org/I1344073410","display_name":"Howard Hughes Medical Institute","ror":"https://ror.org/006w34k90","country_code":"US","type":"facility","lineage":["https://openalex.org/I1344073410"]},{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Katherine A. High","raw_affiliation_strings":["From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","CHILDREN'S HOSPITAL OF PHILADELPHIA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Department of Pediatrics, the Department of Medicine, Hematology/Oncology Division, and the Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine; the Division of Hematology, The Children's Hospital of Philadelphia; and the Howard Hughes Medical Institute, Philadelphia, PA","institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I1344073410","https://openalex.org/I79576946"]},{"raw_affiliation_string":"CHILDREN'S HOSPITAL OF PHILADELPHIA","institution_ids":["https://openalex.org/I1335321130"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":3,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":{"value":5500,"currency":"USD","value_usd":5500},"apc_paid":null,"fwci":2.7182,"has_fulltext":false,"cited_by_count":32,"citation_normalized_percentile":{"value":0.89521014,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":88,"max":97},"biblio":{"volume":"104","issue":"9","first_page":"2767","last_page":"2774"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11280","display_name":"Hemophilia Treatment and Research","score":1.0,"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":1.0,"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":0.0,"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":0.0,"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-b","display_name":"hemophilia B","score":1.0},{"id":"https://openalex.org/keywords/factor-ix","display_name":"factor IX","score":0.7519999742507935},{"id":"https://openalex.org/keywords/mouse-model","display_name":"mouse model","score":0.7770000100135803},{"id":"https://openalex.org/keywords/missense-mutations","display_name":"missense mutations","score":0.7599999904632568},{"id":"https://openalex.org/keywords/premature-termination-codon","display_name":"premature stop codon","score":0.824999988079071},{"id":"https://openalex.org/keywords/gene-deletion","display_name":"gene deletion","score":0.5130000114440918},{"id":"https://openalex.org/keywords/transgenic-mice","display_name":"transgenic mice","score":0.9020000100135803},{"id":"https://openalex.org/keywords/gene-copy-number","display_name":"gene copy number","score":0.6899999976158142},{"id":"https://openalex.org/keywords/liver-expression","display_name":"liver expression","score":0.3880000114440918}],"concepts":[{"id":"https://openalex.org/C2781221834","wikidata":"https://www.wikidata.org/wiki/Q425360","display_name":"Factor IX","level":2,"score":0.9010497331619263},{"id":"https://openalex.org/C75563809","wikidata":"https://www.wikidata.org/wiki/Q2656896","display_name":"Missense mutation","level":4,"score":0.6232691407203674},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.6046633720397949},{"id":"https://openalex.org/C102230213","wikidata":"https://www.wikidata.org/wiki/Q430117","display_name":"Transgene","level":3,"score":0.5842517018318176},{"id":"https://openalex.org/C2908647359","wikidata":"https://www.wikidata.org/wiki/Q2625603","display_name":"Population","level":2,"score":0.5621914863586426},{"id":"https://openalex.org/C501734568","wikidata":"https://www.wikidata.org/wiki/Q42918","display_name":"Mutation","level":3,"score":0.5469253063201904},{"id":"https://openalex.org/C141035611","wikidata":"https://www.wikidata.org/wiki/Q5532934","display_name":"Genetically modified mouse","level":4,"score":0.5191001892089844},{"id":"https://openalex.org/C176944494","wikidata":"https://www.wikidata.org/wiki/Q1415380","display_name":"Point mutation","level":4,"score":0.4978320598602295},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.4714325964450836},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.4354168772697449},{"id":"https://openalex.org/C54009773","wikidata":"https://www.wikidata.org/wiki/Q1429031","display_name":"Transfection","level":3,"score":0.41658827662467957},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.37201759219169617},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.14919307827949524},{"id":"https://openalex.org/C99454951","wikidata":"https://www.wikidata.org/wiki/Q932068","display_name":"Environmental health","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000917","descriptor_name":"Antibody Formation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002836","descriptor_name":"Hemophilia B","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D004195","descriptor_name":"Disease Models, Animal","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D005164","descriptor_name":"Factor IX","qualifier_ui":"Q000032","qualifier_name":"analysis","is_major_topic":false},{"descriptor_ui":"D005164","descriptor_name":"Factor IX","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D005164","descriptor_name":"Factor IX","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008099","descriptor_name":"Liver","qualifier_ui":"Q000737","qualifier_name":"chemistry","is_major_topic":false},{"descriptor_ui":"D008822","descriptor_name":"Mice, Transgenic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009154","descriptor_name":"Mutation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D012313","descriptor_name":"RNA","qualifier_ui":"Q000032","qualifier_name":"analysis","is_major_topic":false},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018389","descriptor_name":"Codon, Nonsense","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D020125","descriptor_name":"Mutation, Missense","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":2,"locations":[{"id":"doi:10.1182/blood-2004-03-1028","doi":"https://doi.org/10.1182/blood-2004-03-1028","is_oa":false,"landing_page_url":"https://doi.org/10.1182/blood-2004-03-1028","pdf_url":null,"source":{"id":"https://openalex.org/S200071133","display_name":"Blood","issn_l":"0006-4971","issn":["0006-4971","1528-0020"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"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":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood","raw_type":"journal-article"},{"id":"pmid:15217833","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/15217833","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","raw_type":"Journal Article"}],"best_oa_location":null,"sustainable_development_goals":[{"score":0.9763000011444092,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"sustainable_development_goals_aurora":[{"score":0.6899999976158142,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G5758617883","display_name":"Gene Therapy for Hemophilia","funder_award_id":"U01HL066948","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G6023877185","display_name":"Gene Therapy for Hemophilia","funder_award_id":"P01HL064190","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G7921163993","display_name":null,"funder_award_id":"R01A1/HL51390","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G7973508433","display_name":"Efficacy and Safety of AAV Gene Transfer for Hemophilia","funder_award_id":"K01DK060580","funder_id":"https://openalex.org/F4320337357","funder_display_name":"National Institute of Diabetes and Digestive and Kidney Diseases"}],"funders":[{"id":"https://openalex.org/F4320337338","display_name":"National Heart, Lung, and Blood Institute","ror":"https://ror.org/012pb6c26"},{"id":"https://openalex.org/F4320337357","display_name":"National Institute of Diabetes and Digestive and Kidney Diseases","ror":"https://ror.org/00adh9b73"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":56,"referenced_works":["https://openalex.org/W30338701","https://openalex.org/W67249329","https://openalex.org/W148295968","https://openalex.org/W149877699","https://openalex.org/W1255418244","https://openalex.org/W1426203004","https://openalex.org/W1484163259","https://openalex.org/W1487084354","https://openalex.org/W1524957948","https://openalex.org/W1588659566","https://openalex.org/W1600331093","https://openalex.org/W1801228818","https://openalex.org/W1964975805","https://openalex.org/W1971011130","https://openalex.org/W1972852573","https://openalex.org/W1978519692","https://openalex.org/W1982328622","https://openalex.org/W1984096039","https://openalex.org/W1984611415","https://openalex.org/W1986747192","https://openalex.org/W1991103963","https://openalex.org/W1993592526","https://openalex.org/W2005059927","https://openalex.org/W2009006613","https://openalex.org/W2011891951","https://openalex.org/W2022441262","https://openalex.org/W2029399500","https://openalex.org/W2036877355","https://openalex.org/W2046740401","https://openalex.org/W2046809665","https://openalex.org/W2047790770","https://openalex.org/W2049592320","https://openalex.org/W2052823768","https://openalex.org/W2071226544","https://openalex.org/W2089745978","https://openalex.org/W2092386839","https://openalex.org/W2093780808","https://openalex.org/W2114708543","https://openalex.org/W2120906365","https://openalex.org/W2122874617","https://openalex.org/W2139691229","https://openalex.org/W2147778207","https://openalex.org/W2149925405","https://openalex.org/W2155710930","https://openalex.org/W2158010208","https://openalex.org/W2159397356","https://openalex.org/W2160400422","https://openalex.org/W2170341515","https://openalex.org/W2269518937","https://openalex.org/W2295080980","https://openalex.org/W2325885736","https://openalex.org/W2337290468","https://openalex.org/W2417696606","https://openalex.org/W2567819392","https://openalex.org/W4252200392","https://openalex.org/W4324114561"],"related_works":["https://openalex.org/W2067695390","https://openalex.org/W2011779126","https://openalex.org/W2269518937","https://openalex.org/W1255418244","https://openalex.org/W1991822046","https://openalex.org/W1497266824","https://openalex.org/W67249329","https://openalex.org/W2149925405","https://openalex.org/W2110175465","https://openalex.org/W1969064455"],"abstract_inverted_index":{"Animal":[0],"models":[1,18,180],"have":[2],"been":[3],"critical":[4],"to":[5,28,168],"the":[6,40,53,62,70,93,96,111,122,134,148,169,174,183],"development":[7],"of":[8,15,19,33,50,64,95,110,130,165],"novel":[9,194],"therapeutics":[10],"in":[11,39,52,69,121,133],"hemophilia.":[12,197],"A":[13],"deficiency":[14],"current":[16],"murine":[17,97,112],"hemophilia":[20,42],"B":[21],"is":[22,36],"that":[23,35,66],"they":[24],"are":[25],"all":[26],"due":[27],"gene":[29],"deletions,":[30],"a":[31,48,107,156,163],"type":[32],"mutation":[34],"relatively":[37],"rare":[38],"human":[41,71,78,186],"population.":[43,72],"We":[44],"generated":[45,101,142],"mice":[46,74,105,135],"with":[47,84,104,140,155],"range":[49,164],"mutations":[51,65,184],"Factor":[54],"IX":[55],"(F.IX)":[56],"gene;":[57],"these":[58],"more":[59],"faithfully":[60,181],"reflect":[61],"types":[63],"cause":[67],"disease":[68],"Transgenic":[73],"expressing":[75,159],"either":[76],"wild-type":[77],"F.IX":[79,82,113,118,131,150],"(hF.IX),":[80],"or":[81,89],"variants":[83],"premature":[85],"translation":[86],"termination":[87],"codons,":[88],"missense":[90],"mutations,":[91],"under":[92],"control":[94],"transthyretin":[98],"promoter,":[99],"were":[100,136,153],"and":[102,127,138,161,188],"crossed":[103],"carrying":[106],"large":[108],"deletion":[109],"gene.":[114],"Gene":[115],"copy":[116],"number,":[117],"transcript":[119],"levels":[120,129],"liver,":[123],"intrahepatocyte":[124],"protein":[125,132],"expression,":[126],"circulating":[128],"determined":[137],"compared":[139],"data":[141],"by":[143],"transient":[144],"transfection":[145],"assays":[146],"using":[147],"same":[149],"variants.":[151],"Mice":[152],"injected":[154],"viral":[157],"vector":[158],"hF.IX":[160],"displayed":[162],"immune":[166],"responses":[167],"transgene":[170],"product,":[171],"depending":[172],"on":[173],"underlying":[175],"mutation.":[176],"These":[177],"new":[178],"mouse":[179],"mimic":[182],"causing":[185],"disease,":[187],"will":[189],"prove":[190],"useful":[191],"for":[192,196],"testing":[193],"therapies":[195]},"counts_by_year":[{"year":2025,"cited_by_count":1},{"year":2024,"cited_by_count":1},{"year":2023,"cited_by_count":1},{"year":2021,"cited_by_count":1},{"year":2019,"cited_by_count":3},{"year":2018,"cited_by_count":1},{"year":2017,"cited_by_count":1},{"year":2015,"cited_by_count":1},{"year":2014,"cited_by_count":2},{"year":2013,"cited_by_count":1}],"updated_date":"2026-11-01T11:40:01.658920","created_date":"2016-06-24T00:00:00"}