{"id":"https://openalex.org/W2015192135","doi":"https://doi.org/10.1371/journal.pone.0013424","title":"Gene Therapy in a Humanized Mouse Model of Familial Hypercholesterolemia Leads to Marked Regression of Atherosclerosis","display_name":"Gene Therapy in a Humanized Mouse Model of Familial Hypercholesterolemia Leads to Marked Regression of Atherosclerosis","publication_year":2010,"publication_date":"2010-10-19","ids":{"openalex":"https://openalex.org/W2015192135","doi":"https://doi.org/10.1371/journal.pone.0013424","mag":"2015192135","pmid":"https://pubmed.ncbi.nlm.nih.gov/20976059","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/2957433"},"language":"en","primary_location":{"id":"doi:10.1371/journal.pone.0013424","doi":"https://doi.org/10.1371/journal.pone.0013424","is_oa":true,"landing_page_url":"https://doi.org/10.1371/journal.pone.0013424","pdf_url":"https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0013424&type=printable","source":{"id":"https://openalex.org/S202381698","display_name":"PLoS ONE","issn_l":"1932-6203","issn":["1932-6203"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-class-a","anvur-scientific","cwts-core","doaj","dongbi-c","doyens","erih-plus","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310315706","host_organization_name":"Public Library of Science","host_organization_lineage":["https://openalex.org/P4310315706"],"host_organization_lineage_names":["Public Library of Science"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"PLoS ONE","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0013424&type=printable","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5060647076","display_name":"Sadik H. Kassim","orcid":"https://orcid.org/0009-0001-3764-3128","observed_orcids":["https://orcid.org/0009-0001-3764-3128"]},"institutions":[{"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":"Sadik H. Kassim","raw_affiliation_strings":["Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5100753259","display_name":"Hui Li","orcid":"https://orcid.org/0000-0003-0589-0359","observed_orcids":["https://orcid.org/0000-0003-0589-0359"]},"institutions":[{"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":"Hui Li","raw_affiliation_strings":["Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5009839456","display_name":"Luk H. Vandenberghe","orcid":"https://orcid.org/0000-0002-3508-4924","observed_orcids":["https://orcid.org/0000-0002-3508-4924"]},"institutions":[{"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":"Luk H. Vandenberghe","raw_affiliation_strings":["Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5046433989","display_name":"Christian J. Hinderer","orcid":null,"observed_orcids":[]},"institutions":[{"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":"Christian Hinderer","raw_affiliation_strings":["Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5083739731","display_name":"Peter L. Bell","orcid":"https://orcid.org/0000-0002-5420-4477","observed_orcids":["https://orcid.org/0000-0002-5420-4477"]},"institutions":[{"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 Bell","raw_affiliation_strings":["Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5083757642","display_name":"Dawn H.L. Marchadier","orcid":null,"observed_orcids":[]},"institutions":[{"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":"Dawn Marchadier","raw_affiliation_strings":["Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5089992789","display_name":"Aisha Wilson","orcid":null,"observed_orcids":[]},"institutions":[{"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":"Aisha Wilson","raw_affiliation_strings":["Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5036959612","display_name":"Debra A. Cromley","orcid":null,"observed_orcids":[]},"institutions":[{"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":"Debra Cromley","raw_affiliation_strings":["Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5033377809","display_name":"Valeska Redon","orcid":null,"observed_orcids":[]},"institutions":[{"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":"Valeska Redon","raw_affiliation_strings":["Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5020162592","display_name":"Hongwei Yu","orcid":"https://orcid.org/0000-0003-3029-3644","observed_orcids":["https://orcid.org/0000-0003-3029-3644"]},"institutions":[{"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":"Hongwei Yu","raw_affiliation_strings":["Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5051344823","display_name":"James M. Wilson","orcid":"https://orcid.org/0000-0002-9630-3131","observed_orcids":["https://orcid.org/0000-0002-9630-3131","https://orcid.org/0000-0002-7988-0201"]},"institutions":[{"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":"James M. Wilson","raw_affiliation_strings":["Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5067149045","display_name":"Daniel James Rader","orcid":"https://orcid.org/0000-0002-9245-9876","observed_orcids":["https://orcid.org/0000-0002-9245-9876"]},"institutions":[{"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":"Daniel J. Rader","raw_affiliation_strings":["Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America","institution_ids":["https://openalex.org/I79576946"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":["https://openalex.org/A5067149045"],"corresponding_institution_ids":["https://openalex.org/I79576946"],"apc_list":{"value":2382,"currency":"USD","value_usd":2382},"apc_paid":{"value":2382,"currency":"USD","value_usd":2382},"fwci":2.6402,"has_fulltext":true,"cited_by_count":81,"citation_normalized_percentile":{"value":0.90549343,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":89,"max":99},"biblio":{"volume":"5","issue":"10","first_page":"e13424","last_page":"e13424"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9380000233650208,"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":0.9380000233650208,"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/T10394","display_name":"Lipoproteins and Cardiovascular Health","score":0.0284000001847744,"subfield":{"id":"https://openalex.org/subfields/2705","display_name":"Cardiology and Cardiovascular Medicine"},"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/T11618","display_name":"Cholesterol and Lipid Metabolism","score":0.013000000268220901,"subfield":{"id":"https://openalex.org/subfields/1303","display_name":"Biochemistry"},"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/familial-hypercholesterolemia","display_name":"familial hypercholesterolemia","score":0.9810000061988831},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.9039999842643738},{"id":"https://openalex.org/keywords/atherosclerosis-regression","display_name":"atherosclerosis regression","score":0.7960000038146973},{"id":"https://openalex.org/keywords/ldl-receptor","display_name":"LDL receptor","score":0.8799999952316284},{"id":"https://openalex.org/keywords/aav8-vector","display_name":"AAV8 vector","score":0.8109999895095825},{"id":"https://openalex.org/keywords/plasma-cholesterol-reduction","display_name":"plasma cholesterol reduction","score":0.5669999718666077},{"id":"https://openalex.org/keywords/western-diet","display_name":"Western diet","score":0.6869999766349792},{"id":"https://openalex.org/keywords/liver-directed-gene-delivery","display_name":"liver-directed gene delivery","score":0.6790000200271606}],"concepts":[{"id":"https://openalex.org/C2779120738","wikidata":"https://www.wikidata.org/wiki/Q2711291","display_name":"Familial hypercholesterolemia","level":3,"score":0.9118507504463196},{"id":"https://openalex.org/C43554185","wikidata":"https://www.wikidata.org/wiki/Q425649","display_name":"LDL receptor","level":4,"score":0.8932482004165649},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.710690438747406},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.5439088940620422},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.519111156463623},{"id":"https://openalex.org/C134018914","wikidata":"https://www.wikidata.org/wiki/Q162606","display_name":"Endocrinology","level":1,"score":0.5111284852027893},{"id":"https://openalex.org/C2778163477","wikidata":"https://www.wikidata.org/wiki/Q43656","display_name":"Cholesterol","level":2,"score":0.4928007423877716},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.3832179009914398},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.3481939435005188},{"id":"https://openalex.org/C2780072125","wikidata":"https://www.wikidata.org/wiki/Q28350","display_name":"Lipoprotein","level":3,"score":0.3366106152534485},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.15277817845344543}],"mesh":[{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D001483","descriptor_name":"Base Sequence","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004195","descriptor_name":"Disease Models, Animal","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006938","descriptor_name":"Hyperlipoproteinemia Type II","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":true},{"descriptor_ui":"D007150","descriptor_name":"Immunohistochemistry","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008810","descriptor_name":"Mice, Inbred C57BL","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D011973","descriptor_name":"Receptors, LDL","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D017931","descriptor_name":"DNA Primers","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D050197","descriptor_name":"Atherosclerosis","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":true},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":5,"locations":[{"id":"doi:10.1371/journal.pone.0013424","doi":"https://doi.org/10.1371/journal.pone.0013424","is_oa":true,"landing_page_url":"https://doi.org/10.1371/journal.pone.0013424","pdf_url":"https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0013424&type=printable","source":{"id":"https://openalex.org/S202381698","display_name":"PLoS ONE","issn_l":"1932-6203","issn":["1932-6203"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-class-a","anvur-scientific","cwts-core","doaj","dongbi-c","doyens","erih-plus","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310315706","host_organization_name":"Public Library of Science","host_organization_lineage":["https://openalex.org/P4310315706"],"host_organization_lineage_names":["Public Library of Science"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"PLoS ONE","raw_type":"journal-article"},{"id":"pmid:20976059","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/20976059","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":"PloS one","raw_type":"Journal Article"},{"id":"pmh:oai:pubmedcentral.nih.gov:2957433","doi":null,"is_oa":true,"landing_page_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC2957433/","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","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"PLoS One","raw_type":"Text"},{"id":"pmh:oai:CiteSeerX.psu:10.1.1.350.6315","doi":null,"is_oa":false,"landing_page_url":"http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.350.6315","pdf_url":null,"source":null,"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"ftp://ftp.ncbi.nlm.nih.gov/pub/pmc/f6/c9/PLoS_One_2010_Oct_19_5(10)_e13424.tar.gz","raw_type":"text"},{"id":"pmh:oai:doaj.org/article:e79587d53a7b4820b632c5dfd2a0f483","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/e79587d53a7b4820b632c5dfd2a0f483","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":"PLoS ONE, Vol 5, Iss 10, p e13424 (2010)","raw_type":"article"}],"best_oa_location":{"id":"doi:10.1371/journal.pone.0013424","doi":"https://doi.org/10.1371/journal.pone.0013424","is_oa":true,"landing_page_url":"https://doi.org/10.1371/journal.pone.0013424","pdf_url":"https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0013424&type=printable","source":{"id":"https://openalex.org/S202381698","display_name":"PLoS ONE","issn_l":"1932-6203","issn":["1932-6203"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-class-a","anvur-scientific","cwts-core","doaj","dongbi-c","doyens","erih-plus","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310315706","host_organization_name":"Public Library of Science","host_organization_lineage":["https://openalex.org/P4310315706"],"host_organization_lineage_names":["Public Library of Science"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"PLoS ONE","raw_type":"journal-article"},"sustainable_development_goals":[{"score":1.0,"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G3682178382","display_name":"Molecular Therapy for Cystic Fibrosis","funder_award_id":"P30DK047757","funder_id":"https://openalex.org/F4320337357","funder_display_name":"National Institute of Diabetes and Digestive and Kidney Diseases"},{"id":"https://openalex.org/G6412557770","display_name":"AAV Gene Therapy for Lipid Disorders","funder_award_id":"P01HL059407","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"}],"funders":[{"id":"https://openalex.org/F4320309370","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10"},{"id":"https://openalex.org/F4320310847","display_name":"University of South Florida","ror":"https://ror.org/032db5x82"},{"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":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W2015192135.pdf","grobid_xml":"https://content.openalex.org/works/W2015192135.grobid-xml"},"referenced_works_count":51,"referenced_works":["https://openalex.org/W1552687651","https://openalex.org/W1585682629","https://openalex.org/W1808026750","https://openalex.org/W1965696997","https://openalex.org/W1967882987","https://openalex.org/W1971716779","https://openalex.org/W1976329054","https://openalex.org/W1977044163","https://openalex.org/W1985656844","https://openalex.org/W1990522102","https://openalex.org/W1990884632","https://openalex.org/W2000185853","https://openalex.org/W2009968900","https://openalex.org/W2010416499","https://openalex.org/W2014364223","https://openalex.org/W2019318413","https://openalex.org/W2019345011","https://openalex.org/W2023133801","https://openalex.org/W2026126653","https://openalex.org/W2027602469","https://openalex.org/W2032435016","https://openalex.org/W2037150431","https://openalex.org/W2037832608","https://openalex.org/W2046488302","https://openalex.org/W2047608515","https://openalex.org/W2048382073","https://openalex.org/W2057548071","https://openalex.org/W2062723580","https://openalex.org/W2062926042","https://openalex.org/W2064240353","https://openalex.org/W2065372271","https://openalex.org/W2076522499","https://openalex.org/W2080951809","https://openalex.org/W2085189052","https://openalex.org/W2088086799","https://openalex.org/W2091918904","https://openalex.org/W2114918865","https://openalex.org/W2115598881","https://openalex.org/W2127014028","https://openalex.org/W2138466097","https://openalex.org/W2141907642","https://openalex.org/W2142108003","https://openalex.org/W2143479464","https://openalex.org/W2166797660","https://openalex.org/W2167414337","https://openalex.org/W2170205772","https://openalex.org/W2183914204","https://openalex.org/W2308510141","https://openalex.org/W2314705141","https://openalex.org/W2316791059","https://openalex.org/W4211221606"],"related_works":["https://openalex.org/W2054925982","https://openalex.org/W4401599845","https://openalex.org/W2051282213","https://openalex.org/W1998463000","https://openalex.org/W2143344248","https://openalex.org/W2563034654","https://openalex.org/W7163572565","https://openalex.org/W4393144020","https://openalex.org/W2127014028","https://openalex.org/W2106369276"],"abstract_inverted_index":{"BACKGROUND:":[0],"Familial":[1],"hypercholesterolemia":[2,25],"(FH)":[3],"is":[4],"an":[5,150],"autosomal":[6],"codominant":[7],"disorder":[8],"caused":[9],"by":[10],"mutations":[11],"in":[12,31,40,105,135],"the":[13,41,76,83,174,204,211],"low-density":[14],"lipoprotein":[15],"receptor":[16],"(LDLR)":[17],"gene.":[18],"Homozygous":[19],"FH":[20,184],"patients":[21],"(hoFH)":[22],"have":[23],"severe":[24],"leading":[26],"to":[27,74,82,96,160],"life":[28],"threatening":[29],"atherosclerosis":[30,136],"childhood":[32],"and":[33,43,49,101,123,188],"adolescence.":[34],"Mice":[35],"with":[36,125,141,147],"germ":[37],"line":[38],"interruptions":[39],"Ldlr":[42,80],"Apobec1":[44],"genes":[45],"(Ldlr(-/-)Apobec1(-/-))":[46],"simulate":[47],"metabolic":[48],"clinical":[50],"aspects":[51],"of":[52,85,92,153,169,192,207,215],"hoFH,":[53],"including":[54],"atherogenesis":[55],"on":[56,67],"a":[57,120,126,131,166],"chow":[58],"diet.":[59],"METHODS/PRINCIPAL":[60],"FINDINGS:":[61],"In":[62],"this":[63,193],"study,":[64],"vectors":[65],"based":[66],"adeno-associated":[68],"virus":[69],"8":[70],"(AAV8)":[71],"were":[72],"used":[73],"deliver":[75],"gene":[77,181,208],"for":[78,183,203,213],"mouse":[79],"(mLDLR)":[81],"livers":[84],"Ldlr(-/-)Apobec1(-/-)":[86,117,144],"mice.":[87,162],"A":[88],"single":[89],"intravenous":[90],"injection":[91],"AAV8.mLDLR":[93,148],"was":[94],"found":[95],"significantly":[97],"reduce":[98],"plasma":[99],"cholesterol":[100,103],"non-HDL":[102],"levels":[104],"chow-fed":[106],"animals":[107],"at":[108],"doses":[109],"as":[110,112],"low":[111],"3\u00d710(9)":[113],"genome":[114],"copies/mouse.":[115],"Whereas":[116],"mice":[118,145],"fed":[119],"western-type":[121],"diet":[122],"injected":[124],"control":[127],"AAV8.null":[128],"vector":[129],"experienced":[130],"further":[132,190],"65%":[133],"progression":[134],"over":[137],"2":[138],"months":[139,158],"compared":[140,159],"baseline":[142,161],"mice,":[143],"treated":[146],"realized":[149],"87%":[151],"regression":[152],"atherosclerotic":[154,170],"lesions":[155],"after":[156],"3":[157],"Immunohistochemical":[163],"analyses":[164],"revealed":[165],"substantial":[167],"remodeling":[168],"lesions.":[171],"CONCLUSIONS/SIGNIFICANCE:":[172],"Collectively,":[173],"results":[175],"presented":[176],"herein":[177],"suggest":[178],"that":[179],"AAV8-based":[180],"therapy":[182,209],"may":[185],"be":[186],"feasible":[187],"support":[189],"development":[191],"approach.":[194],"The":[195],"pre-clinical":[196],"data":[197],"from":[198],"these":[199],"studies":[200],"will":[201],"enable":[202],"effective":[205],"translation":[206],"into":[210],"clinic":[212],"treatment":[214],"FH.":[216]},"counts_by_year":[{"year":2026,"cited_by_count":3},{"year":2025,"cited_by_count":1},{"year":2024,"cited_by_count":3},{"year":2023,"cited_by_count":9},{"year":2022,"cited_by_count":7},{"year":2021,"cited_by_count":2},{"year":2020,"cited_by_count":4},{"year":2019,"cited_by_count":6},{"year":2018,"cited_by_count":2},{"year":2017,"cited_by_count":7},{"year":2016,"cited_by_count":7},{"year":2015,"cited_by_count":7},{"year":2014,"cited_by_count":4},{"year":2013,"cited_by_count":3},{"year":2012,"cited_by_count":6}],"updated_date":"2026-11-12T12:28:57.059296","created_date":"2016-06-24T00:00:00"}
