{"id":"https://openalex.org/W2177849941","doi":"https://doi.org/10.1182/blood.v91.12.4600","title":"Adeno-Associated Viral Vector-Mediated Gene Transfer of Human Blood Coagulation Factor IX Into Mouse Liver","display_name":"Adeno-Associated Viral Vector-Mediated Gene Transfer of Human Blood Coagulation Factor IX Into Mouse Liver","publication_year":1998,"publication_date":"1998-06-15","ids":{"openalex":"https://openalex.org/W2177849941","doi":"https://doi.org/10.1182/blood.v91.12.4600","mag":"2177849941","pmid":"https://pubmed.ncbi.nlm.nih.gov/9616156"},"language":"en","primary_location":{"id":"doi:10.1182/blood.v91.12.4600","doi":"https://doi.org/10.1182/blood.v91.12.4600","is_oa":false,"landing_page_url":"https://doi.org/10.1182/blood.v91.12.4600","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/A5011138455","display_name":"Hiroyuki Nakai","orcid":"https://orcid.org/0000-0002-4213-3130","observed_orcids":["https://orcid.org/0000-0002-4213-3130"]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"Hiroyuki Nakai","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","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/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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 Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5113520015","display_name":"J. Nathan Hagstrom","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"J. Nathan Hagstrom","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Johannes Walter","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"Johannes Walter","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5082877210","display_name":"Szu\u2010Hao Kung","orcid":"https://orcid.org/0000-0002-0674-951X","observed_orcids":["https://orcid.org/0000-0002-0674-951X"]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"Szu-Hao Kung","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5102136137","display_name":"Edmund Y. Yang","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"Edmund Y. Yang","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5024370985","display_name":"Shing Jen Tai","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"Shing Jen Tai","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5111750390","display_name":"Yuichi Iwaki","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"Yuichi Iwaki","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5111597341","display_name":"Gary J. Kurtzman","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"Gary J. Kurtzman","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5113903945","display_name":"Krishna J. Fisher","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"Krishna J. Fisher","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5110021583","display_name":"Peter Colosi","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"Peter Colosi","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5105939178","display_name":"Linda B. Couto","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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":"Linda B. Couto","raw_affiliation_strings":["From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","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/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"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/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 Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From Avigen, Inc, Alameda, CA; the Department of Urology, University of Southern California School of Medicine, Los Angeles, CA; the Departments of Pediatrics, Pathology, and Molecular and Cellular Engineering, University of Pennsylvania School of Medicine, Philadelphia, PA; the Department of Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA; and the Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA","institution_ids":["https://openalex.org/I1174212","https://openalex.org/I1335321130","https://openalex.org/I79576946"]}]}],"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":12.1704,"has_fulltext":false,"cited_by_count":187,"citation_normalized_percentile":{"value":0.99152226,"is_in_top_1_percent":true,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":88,"max":99},"biblio":{"volume":"91","issue":"12","first_page":"4600","last_page":"4607"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9804999828338623,"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"}},"topics":[{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9804999828338623,"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/T11280","display_name":"Hemophilia Treatment and Research","score":0.01640000008046627,"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/T10725","display_name":"RNA Interference and Gene Delivery","score":0.0006000000284984708,"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/recombinant-adeno-associated-virus-vectors","display_name":"recombinant adeno-associated virus vectors","score":0.9120000004768372},{"id":"https://openalex.org/keywords/factor-ix","display_name":"factor IX","score":0.6940000057220459},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.5699999928474426},{"id":"https://openalex.org/keywords/liver-directed-gene-transfer","display_name":"liver-directed gene transfer","score":0.7009999752044678},{"id":"https://openalex.org/keywords/hepatocyte-transduction","display_name":"hepatocyte transduction","score":0.8740000128746033},{"id":"https://openalex.org/keywords/cytomegalovirus-promoter","display_name":"cytomegalovirus promoter","score":0.7950000166893005},{"id":"https://openalex.org/keywords/ef1alpha-promoter","display_name":"EF1\u03b1 promoter","score":0.9760000109672546},{"id":"https://openalex.org/keywords/promoter-selection","display_name":"promoter selection","score":0.421999990940094},{"id":"https://openalex.org/keywords/long-term-transgene-expression","display_name":"long-term transgene expression","score":0.722000002861023},{"id":"https://openalex.org/keywords/hemophilia-b","display_name":"hemophilia B","score":0.8510000109672546},{"id":"https://openalex.org/keywords/portal-vein-injection","display_name":"portal vein injection","score":0.6899999976158142}],"concepts":[{"id":"https://openalex.org/C2781221834","wikidata":"https://www.wikidata.org/wiki/Q425360","display_name":"Factor IX","level":2,"score":0.9004369378089905},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.6663227081298828},{"id":"https://openalex.org/C207001950","wikidata":"https://www.wikidata.org/wiki/Q141124","display_name":"In vivo","level":2,"score":0.5434099435806274},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.5343956351280212},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.517006516456604},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.5065990686416626},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.4691440165042877},{"id":"https://openalex.org/C33161422","wikidata":"https://www.wikidata.org/wiki/Q5421712","display_name":"Expression vector","level":4,"score":0.422167032957077},{"id":"https://openalex.org/C54009773","wikidata":"https://www.wikidata.org/wiki/Q1429031","display_name":"Transfection","level":3,"score":0.4166334569454193},{"id":"https://openalex.org/C92087593","wikidata":"https://www.wikidata.org/wiki/Q427389","display_name":"Vector (molecular biology)","level":4,"score":0.41245782375335693},{"id":"https://openalex.org/C159047783","wikidata":"https://www.wikidata.org/wiki/Q7215","display_name":"Virology","level":1,"score":0.41072767972946167},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.22164538502693176},{"id":"https://openalex.org/C150903083","wikidata":"https://www.wikidata.org/wiki/Q7108","display_name":"Biotechnology","level":1,"score":0.0},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.0},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D000229","descriptor_name":"Dependovirus","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D000328","descriptor_name":"Adult","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005164","descriptor_name":"Factor IX","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D005455","descriptor_name":"Fluorescent Antibody Technique","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005786","descriptor_name":"Gene Expression Regulation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008099","descriptor_name":"Liver","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":true},{"descriptor_ui":"D018014","descriptor_name":"Gene Transfer Techniques","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":3,"locations":[{"id":"doi:10.1182/blood.v91.12.4600","doi":"https://doi.org/10.1182/blood.v91.12.4600","is_oa":false,"landing_page_url":"https://doi.org/10.1182/blood.v91.12.4600","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":"doi:10.1182/blood.v91.12.4600.412k22_4600_4607","doi":"https://doi.org/10.1182/blood.v91.12.4600.412k22_4600_4607","is_oa":false,"landing_page_url":"https://doi.org/10.1182/blood.v91.12.4600.412k22_4600_4607","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:9616156","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/9616156","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":[{"id":"https://metadata.un.org/sdg/3","score":0.98580002784729,"display_name":"Good health and well-being"}],"sustainable_development_goals_aurora":[{"id":"https://metadata.un.org/sdg/3","score":0.5400000214576721,"display_name":"Good health and well-being"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G2378215612","display_name":"ACTIVATION OF HEMOSTASIS","funder_award_id":"P50HL054500","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G2603931035","display_name":null,"funder_award_id":"R01 HL53668","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G953457747","display_name":"NOVEL STRATAGIES FOR GENE THERAPY OF HEMOPHILIA B","funder_award_id":"5r01hl053668-03","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"}],"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":{"pdf":false,"grobid_xml":false},"content_urls":null,"referenced_works_count":41,"referenced_works":["https://openalex.org/W114318986","https://openalex.org/W1514924602","https://openalex.org/W1519383882","https://openalex.org/W1542151085","https://openalex.org/W1542198680","https://openalex.org/W1756236371","https://openalex.org/W1968912221","https://openalex.org/W1970705434","https://openalex.org/W1977115071","https://openalex.org/W1978519692","https://openalex.org/W1983728851","https://openalex.org/W1998290936","https://openalex.org/W2004447115","https://openalex.org/W2005059927","https://openalex.org/W2007854533","https://openalex.org/W2014010760","https://openalex.org/W2014580830","https://openalex.org/W2015219383","https://openalex.org/W2019612944","https://openalex.org/W2029426142","https://openalex.org/W2038036971","https://openalex.org/W2049592320","https://openalex.org/W2077803751","https://openalex.org/W2084210336","https://openalex.org/W2092386839","https://openalex.org/W2114126275","https://openalex.org/W2121944814","https://openalex.org/W2133753843","https://openalex.org/W2139784464","https://openalex.org/W2143836588","https://openalex.org/W2147020694","https://openalex.org/W2148902400","https://openalex.org/W2151902657","https://openalex.org/W2161340273","https://openalex.org/W2165815741","https://openalex.org/W2170234372","https://openalex.org/W2289725452","https://openalex.org/W2419259053","https://openalex.org/W2427176710","https://openalex.org/W3167881864","https://openalex.org/W4244944523"],"related_works":["https://openalex.org/W2128684337","https://openalex.org/W1978519692","https://openalex.org/W1976329054","https://openalex.org/W2064150651","https://openalex.org/W2029426142","https://openalex.org/W2173845487","https://openalex.org/W2157900284","https://openalex.org/W1967882987","https://openalex.org/W2138084195","https://openalex.org/W1971911791"],"abstract_inverted_index":{"Recombinant":[0],"adeno-associated":[1],"virus":[2],"vectors":[3],"(AAV)":[4],"were":[5],"prepared":[6],"in":[7,22,46,52,55,74,101,135,141,174,196,213,253],"high":[8],"titer":[9],"(10(12)":[10],"to":[11,69,84,106,179,218],"10(13)":[12],"particles/mL)":[13],"for":[14,182],"the":[15,33,39,62,75,120,148,157,228,263],"expression":[16,65,73,92,113,144,223,248,258],"of":[17,25,29,42,93,103,112,119,132,160,167,185,198,204,207,216,224,249,265],"human":[18,34,49,94,149,192,225],"factor":[19,50],"IX":[20,51,206],"after":[21],"vivo":[23],"transduction":[24],"murine":[26],"hepatocytes.":[27],"Injection":[28],"AAV-CMV-F.IX":[30],"(expression":[31],"from":[32,227],"cytomegalovirus":[35],"IE":[36],"enhancer/promoter)":[37],"into":[38,156],"portal":[40,158],"vein":[41,159],"adult":[43,161],"mice":[44,56,163,199],"resulted":[45,173,212],"no":[47,129,191],"detectable":[48],"plasma,":[53],"but":[54,256],"injected":[57,155,200],"intravenously":[58],"as":[59],"newborns":[60],"with":[61,201],"same":[63],"vector,":[64],"was":[66,77,96,114,145,152,194,231],"initially":[67],"55":[68],"110":[70],"ng/mL.":[71,221],"The":[72,110],"liver":[76],"mostly":[78],"transient,":[79],"and":[80,154,245],"plasma":[81,176,197,214],"levels":[82,86,177,215],"decreased":[83],"undetectable":[85],"within":[87],"5":[88],"weeks.":[89],"However,":[90],"long-term":[91],"F.IX":[95,143,193,226,251],"detected":[97,195],"by":[98,117,147,233],"immunofluorescence":[99,234],"staining":[100],"0.25%":[102],"hepatocytes":[104,244],"8":[105],"10":[107],"months":[108],"postinjection.":[109],"loss":[111,131],"likely":[115],"caused":[116],"suppression":[118],"CMV":[121],"promoter,":[122],"because":[123],"polymerase":[124],"chain":[125],"reaction":[126],"data":[127],"showed":[128],"substantial":[130],"vector":[133,140,230],"DNA":[134],"mouse":[136,254],"liver.":[137],"A":[138],"second":[139],"which":[142],"controlled":[146],"EF1alpha":[150],"promoter":[151],"constructed":[153],"C57BL/6":[162],"at":[164,186],"a":[165,183],"dose":[166],"6.3":[168],"x":[169,209],"10(10)":[170],"particles.":[171],"This":[172],"therapeutic":[175],"(200":[178],"320":[180],"ng/mL)":[181],"period":[184],"least":[187],"6":[188],"months,":[189],"whereas":[190],"AAV-CMV-F.IX.":[202],"Doses":[203],"AAV-EF1alpha-F.":[205],"2.7":[208],"10(11)":[210],"particles":[211],"700":[217],"3,":[219],"200":[220],"Liver-derived":[222],"AAV-EF1alpha-F.IX":[229],"confirmed":[232],"staining.":[235],"We":[236],"conclude":[237],"that":[238],"recombinant":[239],"AAV":[240],"can":[241],"efficiently":[242],"transduce":[243],"direct":[246],"stable":[247],"an":[250],"transgene":[252],"liver,":[255],"sustained":[257],"is":[259],"critically":[260],"dependent":[261],"on":[262],"choice":[264],"promoter.":[266]},"counts_by_year":[{"year":2025,"cited_by_count":5},{"year":2024,"cited_by_count":1},{"year":2023,"cited_by_count":2},{"year":2022,"cited_by_count":3},{"year":2021,"cited_by_count":1},{"year":2020,"cited_by_count":1},{"year":2019,"cited_by_count":3},{"year":2018,"cited_by_count":2},{"year":2017,"cited_by_count":1},{"year":2016,"cited_by_count":1},{"year":2015,"cited_by_count":2},{"year":2014,"cited_by_count":1},{"year":2013,"cited_by_count":7},{"year":2012,"cited_by_count":3}],"updated_date":"2026-11-01T11:40:01.658920","created_date":"2016-06-24T00:00:00"}
