{"id":"https://openalex.org/W3216123748","doi":"https://doi.org/10.1016/j.omtm.2021.11.005","title":"Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A","display_name":"Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A","publication_year":2021,"publication_date":"2021-11-24","ids":{"openalex":"https://openalex.org/W3216123748","doi":"https://doi.org/10.1016/j.omtm.2021.11.005","mag":"3216123748","pmid":"https://pubmed.ncbi.nlm.nih.gov/34977269","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/8666598"},"language":"en","primary_location":{"id":"doi:10.1016/j.omtm.2021.11.005","doi":"https://doi.org/10.1016/j.omtm.2021.11.005","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.omtm.2021.11.005","pdf_url":"http://www.cell.com/article/S2329050121001777/pdf","source":{"id":"https://openalex.org/S2764588101","display_name":"Molecular Therapy \u2014 Methods & Clinical Development","issn_l":"2329-0501","issn":["2329-0501"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-d","ki-jl-1","poland-100","vabb-shw"],"host_organization":"https://openalex.org/P4310315673","host_organization_name":"Cell Press","host_organization_lineage":["https://openalex.org/P4310315673","https://openalex.org/P4310320990"],"host_organization_lineage_names":["Cell Press","Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy - Methods & Clinical Development","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"http://www.cell.com/article/S2329050121001777/pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5028897626","display_name":"Liron Elkouby","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/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Liron Elkouby","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA","The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]},{"raw_affiliation_string":"The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA","institution_ids":["https://openalex.org/I1335321130"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5050063788","display_name":"Sean M. Armour","orcid":"https://orcid.org/0000-0003-4297-4324","observed_orcids":["https://orcid.org/0000-0003-4297-4324"]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Sean M. Armour","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":"https://orcid.org/0000-0003-4297-4324","affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5071866545","display_name":"Raffaella Toso","orcid":"https://orcid.org/0000-0002-1361-7347","observed_orcids":["https://orcid.org/0000-0002-1361-7347"]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Raffaella Toso","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":"https://orcid.org/0000-0002-1361-7347","affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5008307622","display_name":"Marti A. DiPietro","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Marti DiPietro","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Robert J. Davidson","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"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Robert J. Davidson","raw_affiliation_strings":["The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA","institution_ids":["https://openalex.org/I1335321130"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5083116189","display_name":"Giang N. Nguyen","orcid":"https://orcid.org/0000-0003-3205-9106","observed_orcids":["https://orcid.org/0000-0003-3205-9106"]},"institutions":[{"id":"https://openalex.org/I1335321130","display_name":"Children's Hospital of Philadelphia","ror":"https://ror.org/01z7r7q48","country_code":"US","type":"funder","lineage":["https://openalex.org/I1335321130"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Giang N. Nguyen","raw_affiliation_strings":["The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA"],"raw_orcid":"https://orcid.org/0000-0003-3205-9106","affiliations":[{"raw_affiliation_string":"The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA","institution_ids":["https://openalex.org/I1335321130"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5079687223","display_name":"Mallory Willet","orcid":"https://orcid.org/0000-0002-0332-716X","observed_orcids":["https://orcid.org/0000-0002-0332-716X"]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Mallory Willet","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":"https://orcid.org/0000-0002-0332-716X","affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5083415379","display_name":"Stephanie Kutza","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Stephanie Kutza","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5021160966","display_name":"Joseph Silverberg","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Joseph Silverberg","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5045162512","display_name":"Jennifer E. Frick","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jennifer Frick","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5019711358","display_name":"Marco Crosariol","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Marco Crosariol","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5016980817","display_name":"Yuhuan Wang","orcid":"https://orcid.org/0000-0001-6861-6205","observed_orcids":["https://orcid.org/0000-0001-6861-6205"]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Yuhuan Wang","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5005737782","display_name":"Chuansong Wang","orcid":"https://orcid.org/0000-0003-4501-2518","observed_orcids":["https://orcid.org/0000-0003-4501-2518"]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Chuansong Wang","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","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/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Katherine A. High","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]},{"author_position":"middle","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/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":true,"raw_author_name":"Denise E. Sabatino","raw_affiliation_strings":["Division of Hematology, Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA","The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA"],"raw_orcid":"https://orcid.org/0000-0002-6116-5453","affiliations":[{"raw_affiliation_string":"Division of Hematology, Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA","institution_ids":["https://openalex.org/I79576946"]},{"raw_affiliation_string":"The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA","institution_ids":["https://openalex.org/I1335321130"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5010600720","display_name":"Xavier M. Anguela","orcid":"https://orcid.org/0000-0003-2430-8598","observed_orcids":["https://orcid.org/0000-0003-2430-8598"]},"institutions":[{"id":"https://openalex.org/I4210102540","display_name":"Spark Therapeutics (United States)","ror":"https://ror.org/00s93j442","country_code":"US","type":"company","lineage":["https://openalex.org/I4210102540"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Xavier M. Anguela","raw_affiliation_strings":["Spark Therapeutics, Inc., Philadelphia, PA, USA"],"raw_orcid":"https://orcid.org/0000-0003-2430-8598","affiliations":[{"raw_affiliation_string":"Spark Therapeutics, Inc., Philadelphia, PA, USA","institution_ids":["https://openalex.org/I4210102540"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":3,"corresponding_author_ids":["https://openalex.org/A5007360666","https://openalex.org/A5010600720"],"corresponding_institution_ids":["https://openalex.org/I1335321130","https://openalex.org/I4210102540","https://openalex.org/I79576946"],"apc_list":{"value":3900,"currency":"USD","value_usd":3900},"apc_paid":{"value":3900,"currency":"USD","value_usd":3900},"fwci":1.2722,"has_fulltext":false,"cited_by_count":17,"citation_normalized_percentile":{"value":0.79139031,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":95,"max":98},"biblio":{"volume":"24","issue":null,"first_page":"20","last_page":"29"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.5634999871253967,"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":0.5634999871253967,"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":0.43479999899864197,"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":0.0003000000142492354,"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":0.9440000057220459},{"id":"https://openalex.org/keywords/adeno-associated-virus-vectors","display_name":"adeno-associated virus vectors","score":0.8809999823570251},{"id":"https://openalex.org/keywords/factor-viii","display_name":"factor VIII","score":0.7870000004768372},{"id":"https://openalex.org/keywords/liver-directed-gene-therapy","display_name":"liver-directed gene therapy","score":0.781000018119812},{"id":"https://openalex.org/keywords/b-domain-deleted-factor-viii","display_name":"B-domain-deleted factor VIII","score":0.8429999947547913},{"id":"https://openalex.org/keywords/nonhuman-primates","display_name":"non-human primates","score":0.7289999723434448},{"id":"https://openalex.org/keywords/mice","display_name":"mice","score":0.527999997138977}],"concepts":[{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.835544764995575},{"id":"https://openalex.org/C202878990","wikidata":"https://www.wikidata.org/wiki/Q830465","display_name":"Capsid","level":3,"score":0.8336549997329712},{"id":"https://openalex.org/C2778107364","wikidata":"https://www.wikidata.org/wiki/Q355989","display_name":"Adeno-associated virus","level":5,"score":0.7368967533111572},{"id":"https://openalex.org/C8891405","wikidata":"https://www.wikidata.org/wiki/Q1059","display_name":"Immune system","level":2,"score":0.6725112795829773},{"id":"https://openalex.org/C102230213","wikidata":"https://www.wikidata.org/wiki/Q430117","display_name":"Transgene","level":3,"score":0.6644138097763062},{"id":"https://openalex.org/C92087593","wikidata":"https://www.wikidata.org/wiki/Q427389","display_name":"Vector (molecular biology)","level":4,"score":0.6582666635513306},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.5809634923934937},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.5186785459518433},{"id":"https://openalex.org/C159047783","wikidata":"https://www.wikidata.org/wiki/Q7215","display_name":"Virology","level":1,"score":0.5067275166511536},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.4875011146068573},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.41766759753227234},{"id":"https://openalex.org/C2781221834","wikidata":"https://www.wikidata.org/wiki/Q425360","display_name":"Factor IX","level":2,"score":0.41507527232170105},{"id":"https://openalex.org/C2522874641","wikidata":"https://www.wikidata.org/wiki/Q808","display_name":"Virus","level":2,"score":0.3841996192932129},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.3423009514808655},{"id":"https://openalex.org/C70721500","wikidata":"https://www.wikidata.org/wiki/Q177005","display_name":"Computational biology","level":1,"score":0.33266177773475647},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.31418678164482117},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.2021574079990387},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.08993342518806458}],"mesh":[],"locations_count":4,"locations":[{"id":"doi:10.1016/j.omtm.2021.11.005","doi":"https://doi.org/10.1016/j.omtm.2021.11.005","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.omtm.2021.11.005","pdf_url":"http://www.cell.com/article/S2329050121001777/pdf","source":{"id":"https://openalex.org/S2764588101","display_name":"Molecular Therapy \u2014 Methods & Clinical Development","issn_l":"2329-0501","issn":["2329-0501"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-d","ki-jl-1","poland-100","vabb-shw"],"host_organization":"https://openalex.org/P4310315673","host_organization_name":"Cell Press","host_organization_lineage":["https://openalex.org/P4310315673","https://openalex.org/P4310320990"],"host_organization_lineage_names":["Cell Press","Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy - Methods & Clinical Development","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:8666598","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/8666598","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Mol Ther Methods Clin Dev","raw_type":"Text"},{"id":"pmid:34977269","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/34977269","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":"Molecular therapy. Methods & clinical development","raw_type":"Journal Article"},{"id":"pmh:oai:doaj.org/article:8d15f9b5838e44d49c75621a886fa6f5","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/8d15f9b5838e44d49c75621a886fa6f5","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":"Molecular Therapy: Methods & Clinical Development, Vol 24, Iss , Pp 20-29 (2022)","raw_type":"article"}],"best_oa_location":{"id":"doi:10.1016/j.omtm.2021.11.005","doi":"https://doi.org/10.1016/j.omtm.2021.11.005","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.omtm.2021.11.005","pdf_url":"http://www.cell.com/article/S2329050121001777/pdf","source":{"id":"https://openalex.org/S2764588101","display_name":"Molecular Therapy \u2014 Methods & Clinical Development","issn_l":"2329-0501","issn":["2329-0501"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-d","ki-jl-1","poland-100","vabb-shw"],"host_organization":"https://openalex.org/P4310315673","host_organization_name":"Cell Press","host_organization_lineage":["https://openalex.org/P4310315673","https://openalex.org/P4310320990"],"host_organization_lineage_names":["Cell Press","Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy - Methods & Clinical Development","raw_type":"journal-article"},"sustainable_development_goals":[{"display_name":"Good health and well-being","score":0.98580002784729,"id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G5320602136","display_name":"Novel factor VIII variants for improved efficacy in gene therapy for hemophilia A","funder_award_id":"R01HL126850","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"}],"funders":[{"id":"https://openalex.org/F4320337338","display_name":"National Heart, Lung, and Blood Institute","ror":"https://ror.org/012pb6c26"}],"has_content":{"pdf":true,"grobid_xml":false},"content_urls":{"pdf":"https://content.openalex.org/works/W3216123748.pdf"},"referenced_works_count":39,"referenced_works":["https://openalex.org/W1564299213","https://openalex.org/W1568135308","https://openalex.org/W1964975805","https://openalex.org/W1969271703","https://openalex.org/W1975528602","https://openalex.org/W1976329054","https://openalex.org/W1981950767","https://openalex.org/W1986993620","https://openalex.org/W1990107586","https://openalex.org/W1991453997","https://openalex.org/W1995793532","https://openalex.org/W2043149070","https://openalex.org/W2051442157","https://openalex.org/W2058518705","https://openalex.org/W2058942457","https://openalex.org/W2059988954","https://openalex.org/W2062321149","https://openalex.org/W2067204822","https://openalex.org/W2067691677","https://openalex.org/W2075785803","https://openalex.org/W2104531050","https://openalex.org/W2110881562","https://openalex.org/W2127031749","https://openalex.org/W2153867928","https://openalex.org/W2166492122","https://openalex.org/W2320147764","https://openalex.org/W2337281790","https://openalex.org/W2400075822","https://openalex.org/W2533097147","https://openalex.org/W2603828830","https://openalex.org/W2618968334","https://openalex.org/W2773253161","https://openalex.org/W2773565340","https://openalex.org/W2775538622","https://openalex.org/W3080758726","https://openalex.org/W3087143068","https://openalex.org/W3094918774","https://openalex.org/W3102945151","https://openalex.org/W3211832397"],"related_works":["https://openalex.org/W2907288153","https://openalex.org/W3211832397","https://openalex.org/W1991453997","https://openalex.org/W2567471297","https://openalex.org/W2569051573","https://openalex.org/W4310106510","https://openalex.org/W4310108465","https://openalex.org/W2808199143","https://openalex.org/W2565937776","https://openalex.org/W4389220333"],"abstract_inverted_index":{"Extensive":[0],"clinical":[1,76],"data":[2],"from":[3],"liver-mediated":[4],"gene":[5,133],"therapy":[6],"trials":[7],"have":[8],"shown":[9],"that":[10,83],"dose-dependent":[11],"immune":[12,31,72],"responses":[13],"against":[14],"the":[15,59,66,75,81,85,106,166,170,191,195],"vector":[16,46,62],"capsid":[17,172],"may":[18],"impair":[19],"or":[20,144],"even":[21],"preclude":[22],"transgene":[23],"expression":[24,111],"if":[25],"not":[26],"managed":[27],"successfully":[28],"with":[29,101,137],"prompt":[30],"suppression.":[32],"The":[33],"goal":[34],"of":[35,48,52,69,108,150,158,168,187],"this":[36],"preclinical":[37,163],"study":[38,97],"was":[39,113],"to":[40,64,182],"generate":[41],"an":[42],"adeno-associated":[43],"viral":[44],"(AAV)":[45],"capable":[47],"expressing":[49],"therapeutic":[50,177],"levels":[51,149],"B-domain":[53],"deleted":[54],"factor":[55],"VIII":[56],"(FVIII)":[57],"at":[58],"lowest":[60],"possible":[61],"dose":[63,173,184],"minimize":[65],"potential":[67],"Risk":[68],"a":[70,94,175,183],"capsid-mediated":[71],"response":[73],"in":[74,93,99,115,118,194],"setting.":[77],"Here,":[78],"we":[79],"describe":[80],"studies":[82],"identified":[84],"investigational":[86],"agent":[87],"SPK-8011":[88],",":[89],"currently":[90],"being":[91],"evaluated":[92,114],"phase":[95],"1/2":[96],"(NCT03003533)":[98],"individuals":[100],"hemophilia":[102,180],"A.":[103],"In":[104],"particular,":[105],"potency":[107],"our":[109],"second-generation":[110],"cassettes":[112],"mice":[116],"and":[117,127,154],"non-human":[119],"primates":[120,135],"using":[121],"two":[122],"different":[123],"bioengineered":[124],"capsids":[125],"(AAV-Spark100":[126],"AAV-Spark200).":[128],"At":[129],"2":[130,138],"weeks":[131],"after":[132],"transfer,":[134],"transduced":[136],"\u00d7":[139],"10":[140],"12":[141],"vg/kg":[142],"AAV-Spark100-FVIII":[143],"AAV-Spark200-FVIII":[145],"expressed":[146],"FVIII":[147],"antigen":[148],"13%":[151],"\u00b1":[152,156],"2%":[153],"22%":[155],"6%":[157],"normal,":[159],"respectively.":[160],"Collectively,":[161],"these":[162],"results":[164],"validate":[165],"feasibility":[167],"lowering":[169],"AAV":[171],"for":[174,179],"gene-based":[176],"approach":[178],"A":[181],"level":[185],"orders":[186],"magnitude":[188],"lower":[189],"than":[190],"first-generation":[192],"vectors":[193],"clinic.":[196]},"counts_by_year":[{"year":2026,"cited_by_count":3},{"year":2025,"cited_by_count":5},{"year":2024,"cited_by_count":3},{"year":2023,"cited_by_count":3},{"year":2022,"cited_by_count":3}],"updated_date":"2026-10-28T10:33:24.094632","created_date":"2021-12-06T00:00:00"}