{"id":"https://openalex.org/W4387564437","doi":"https://doi.org/10.1097/hep.0000000000000631","title":"Complete correction of murine phenylketonuria by selection-enhanced hepatocyte transplantation","display_name":"Complete correction of murine phenylketonuria by selection-enhanced hepatocyte transplantation","publication_year":2023,"publication_date":"2023-10-12","ids":{"openalex":"https://openalex.org/W4387564437","doi":"https://doi.org/10.1097/hep.0000000000000631","pmid":"https://pubmed.ncbi.nlm.nih.gov/37693457","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/10491101"},"language":"en","primary_location":{"id":"doi:10.1097/hep.0000000000000631","doi":"https://doi.org/10.1097/hep.0000000000000631","is_oa":false,"landing_page_url":"https://doi.org/10.1097/hep.0000000000000631","pdf_url":null,"source":{"id":"https://openalex.org/S156174673","display_name":"Hepatology","issn_l":"0270-9139","issn":["0270-9139","1527-3350"],"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/P4310315671","host_organization_name":"Lippincott Williams & Wilkins","host_organization_lineage":["https://openalex.org/P4310315671","https://openalex.org/P4310318547"],"host_organization_lineage_names":["Lippincott Williams & Wilkins","Wolters Kluwer"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Hepatology","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"https://www.biorxiv.org/content/biorxiv/early/2023/08/28/2023.08.27.554228.full.pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5027839348","display_name":"Anne Vonada","orcid":"https://orcid.org/0000-0003-0398-1898","observed_orcids":["https://orcid.org/0000-0003-0398-1898"]},"institutions":[{"id":"https://openalex.org/I165690674","display_name":"Oregon Health & Science University","ror":"https://ror.org/009avj582","country_code":"US","type":"education","lineage":["https://openalex.org/I165690674"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Anne Vonada","raw_affiliation_strings":["Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, Oregon, USA","Oregon Stem Cell Center, Oregon Health & Science University, Portland, Oregon, USA","Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR 97239, USA","Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA"],"raw_orcid":"https://orcid.org/0000-0003-0398-1898","affiliations":[{"raw_affiliation_string":"Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Oregon Stem Cell Center, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5008533191","display_name":"Leslie Wakefield","orcid":"https://orcid.org/0000-0001-5623-4285","observed_orcids":["https://orcid.org/0000-0001-5623-4285"]},"institutions":[{"id":"https://openalex.org/I165690674","display_name":"Oregon Health & Science University","ror":"https://ror.org/009avj582","country_code":"US","type":"education","lineage":["https://openalex.org/I165690674"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Leslie Wakefield","raw_affiliation_strings":["Department of Pediatrics, Oregon Health & Science University, Portland, Oregon, USA","Oregon Stem Cell Center, Oregon Health & Science University, Portland, Oregon, USA","Department of Pediatrics, Oregon Health & Science University, Portland, OR 97239, USA","Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA"],"raw_orcid":"https://orcid.org/0000-0001-5623-4285","affiliations":[{"raw_affiliation_string":"Department of Pediatrics, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Oregon Stem Cell Center, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Department of Pediatrics, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5048353499","display_name":"Michael Martinez","orcid":"https://orcid.org/0000-0001-6577-9420","observed_orcids":["https://orcid.org/0000-0001-6577-9420"]},"institutions":[{"id":"https://openalex.org/I165690674","display_name":"Oregon Health & Science University","ror":"https://ror.org/009avj582","country_code":"US","type":"education","lineage":["https://openalex.org/I165690674"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Michael Martinez","raw_affiliation_strings":["Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, Oregon, USA","Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR 97239, USA"],"raw_orcid":"https://orcid.org/0000-0001-6577-9420","affiliations":[{"raw_affiliation_string":"Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5002194433","display_name":"Cary O. Harding","orcid":"https://orcid.org/0000-0001-9146-3112","observed_orcids":["https://orcid.org/0000-0001-9146-3112"]},"institutions":[{"id":"https://openalex.org/I165690674","display_name":"Oregon Health & Science University","ror":"https://ror.org/009avj582","country_code":"US","type":"education","lineage":["https://openalex.org/I165690674"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Cary O. Harding","raw_affiliation_strings":["Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, Oregon, USA","Department of Pediatrics, Oregon Health & Science University, Portland, Oregon, USA","Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR 97239, USA","Department of Pediatrics, Oregon Health & Science University, Portland, OR 97239, USA"],"raw_orcid":"https://orcid.org/0000-0001-9146-3112","affiliations":[{"raw_affiliation_string":"Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Department of Pediatrics, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Department of Pediatrics, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5067712261","display_name":"Markus Grompe","orcid":"https://orcid.org/0000-0002-6616-4345","observed_orcids":["https://orcid.org/0000-0002-6616-4345"]},"institutions":[{"id":"https://openalex.org/I165690674","display_name":"Oregon Health & Science University","ror":"https://ror.org/009avj582","country_code":"US","type":"education","lineage":["https://openalex.org/I165690674"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Markus Grompe","raw_affiliation_strings":["Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, Oregon, USA","Department of Pediatrics, Oregon Health & Science University, Portland, Oregon, USA","Oregon Stem Cell Center, Oregon Health & Science University, Portland, Oregon, USA","Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR 97239, USA","Department of Pediatrics, Oregon Health & Science University, Portland, OR 97239, USA","Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA"],"raw_orcid":"https://orcid.org/0000-0002-6616-4345","affiliations":[{"raw_affiliation_string":"Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Department of Pediatrics, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Oregon Stem Cell Center, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Department of Pediatrics, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5040509783","display_name":"Amita Tiyaboonchai","orcid":"https://orcid.org/0000-0002-0116-7393","observed_orcids":["https://orcid.org/0000-0002-0116-7393"]},"institutions":[{"id":"https://openalex.org/I165690674","display_name":"Oregon Health & Science University","ror":"https://ror.org/009avj582","country_code":"US","type":"education","lineage":["https://openalex.org/I165690674"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Amita Tiyaboonchai","raw_affiliation_strings":["Department of Pediatrics, Oregon Health & Science University, Portland, Oregon, USA","Oregon Stem Cell Center, Oregon Health & Science University, Portland, Oregon, USA","Department of Pediatrics, Oregon Health & Science University, Portland, OR 97239, USA","Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA"],"raw_orcid":"https://orcid.org/0000-0002-0116-7393","affiliations":[{"raw_affiliation_string":"Department of Pediatrics, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Oregon Stem Cell Center, Oregon Health & Science University, Portland, Oregon, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Department of Pediatrics, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]},{"raw_affiliation_string":"Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA","institution_ids":["https://openalex.org/I165690674"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":["https://openalex.org/A5027839348"],"corresponding_institution_ids":["https://openalex.org/I165690674"],"apc_list":{"value":2900,"currency":"USD","value_usd":2900},"apc_paid":null,"fwci":3.6933,"has_fulltext":true,"cited_by_count":19,"citation_normalized_percentile":{"value":0.94165316,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":89,"max":99},"biblio":{"volume":"79","issue":"5","first_page":"1088","last_page":"1097"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11027","display_name":"Metabolism and Genetic Disorders","score":0.24690000712871552,"subfield":{"id":"https://openalex.org/subfields/1308","display_name":"Clinical 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"}},"topics":[{"id":"https://openalex.org/T11027","display_name":"Metabolism and Genetic Disorders","score":0.24690000712871552,"subfield":{"id":"https://openalex.org/subfields/1308","display_name":"Clinical 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"}},{"id":"https://openalex.org/T10966","display_name":"Liver physiology and pathology","score":0.17110000550746918,"subfield":{"id":"https://openalex.org/subfields/2721","display_name":"Hepatology"},"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/T11465","display_name":"Drug-Induced Hepatotoxicity and Protection","score":0.10540000349283218,"subfield":{"id":"https://openalex.org/subfields/3004","display_name":"Pharmacology"},"field":{"id":"https://openalex.org/fields/30","display_name":"Pharmacology, Toxicology and Pharmaceutics"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/phenylketonuria","display_name":"phenylketonuria","score":0.9990000128746033},{"id":"https://openalex.org/keywords/hepatocyte-transplantation","display_name":"hepatocyte transplantation","score":0.9800000190734863},{"id":"https://openalex.org/keywords/phenylalanine-metabolism","display_name":"phenylalanine metabolism","score":0.6489999890327454},{"id":"https://openalex.org/keywords/liver-regeneration","display_name":"liver regeneration","score":0.5120000243186951},{"id":"https://openalex.org/keywords/genetic-liver-disease","display_name":"hereditary liver disease","score":0.5730000138282776},{"id":"https://openalex.org/keywords/mouse-model","display_name":"mouse model","score":0.5120000243186951}],"concepts":[{"id":"https://openalex.org/C2776200302","wikidata":"https://www.wikidata.org/wiki/Q827450","display_name":"Hepatocyte","level":3,"score":0.7399306893348694},{"id":"https://openalex.org/C2778722691","wikidata":"https://www.wikidata.org/wiki/Q57055","display_name":"Acetaminophen","level":2,"score":0.6435697078704834},{"id":"https://openalex.org/C2911091166","wikidata":"https://www.wikidata.org/wiki/Q106419912","display_name":"Transplantation","level":2,"score":0.6281267404556274},{"id":"https://openalex.org/C2779609443","wikidata":"https://www.wikidata.org/wiki/Q1368191","display_name":"Liver transplantation","level":3,"score":0.5195184350013733},{"id":"https://openalex.org/C2780497538","wikidata":"https://www.wikidata.org/wiki/Q1192006","display_name":"Detoxification (alternative medicine)","level":3,"score":0.4850555956363678},{"id":"https://openalex.org/C102230213","wikidata":"https://www.wikidata.org/wiki/Q430117","display_name":"Transgene","level":3,"score":0.43564313650131226},{"id":"https://openalex.org/C81917197","wikidata":"https://www.wikidata.org/wiki/Q628760","display_name":"Selection (genetic algorithm)","level":2,"score":0.427173376083374},{"id":"https://openalex.org/C502942594","wikidata":"https://www.wikidata.org/wiki/Q3421914","display_name":"Cancer research","level":1,"score":0.3960195779800415},{"id":"https://openalex.org/C98274493","wikidata":"https://www.wikidata.org/wiki/Q128406","display_name":"Pharmacology","level":1,"score":0.39028656482696533},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.37861838936805725},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.35043269395828247},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.2990204393863678},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.2219116985797882},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.19224968552589417},{"id":"https://openalex.org/C202751555","wikidata":"https://www.wikidata.org/wiki/Q221681","display_name":"In vitro","level":2,"score":0.16260844469070435},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.12266528606414795},{"id":"https://openalex.org/C41008148","wikidata":"https://www.wikidata.org/wiki/Q21198","display_name":"Computer science","level":0,"score":0.06645721197128296},{"id":"https://openalex.org/C204787440","wikidata":"https://www.wikidata.org/wiki/Q188504","display_name":"Alternative medicine","level":2,"score":0.0},{"id":"https://openalex.org/C154945302","wikidata":"https://www.wikidata.org/wiki/Q11660","display_name":"Artificial intelligence","level":1,"score":0.0}],"mesh":[],"locations_count":6,"locations":[{"id":"doi:10.1097/hep.0000000000000631","doi":"https://doi.org/10.1097/hep.0000000000000631","is_oa":false,"landing_page_url":"https://doi.org/10.1097/hep.0000000000000631","pdf_url":null,"source":{"id":"https://openalex.org/S156174673","display_name":"Hepatology","issn_l":"0270-9139","issn":["0270-9139","1527-3350"],"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/P4310315671","host_organization_name":"Lippincott Williams & Wilkins","host_organization_lineage":["https://openalex.org/P4310315671","https://openalex.org/P4310318547"],"host_organization_lineage_names":["Lippincott Williams & Wilkins","Wolters Kluwer"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Hepatology","raw_type":"journal-article"},{"id":"doi:10.1101/2023.08.27.554228","doi":"https://doi.org/10.1101/2023.08.27.554228","is_oa":true,"landing_page_url":"https://doi.org/10.1101/2023.08.27.554228","pdf_url":"https://www.biorxiv.org/content/biorxiv/early/2023/08/28/2023.08.27.554228.full.pdf","source":{"id":"https://openalex.org/S4306402567","display_name":"bioRxiv (Cold Spring Harbor Laboratory)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2750212522","host_organization_name":"Cold Spring Harbor Laboratory","host_organization_lineage":["https://openalex.org/I2750212522"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"bioRxiv","raw_type":"posted-content"},{"id":"pmid:37693457","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/37693457","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":"bioRxiv : the preprint server for biology","raw_type":"Preprint"},{"id":"pmid:37824086","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/37824086","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":"Hepatology (Baltimore, Md.)","raw_type":"Journal Article"},{"id":"pmh:oai:pubmedcentral.nih.gov:11559556","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/11559556","pdf_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC11559556/pdf/nihms-2032397.pdf","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":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"Hepatology","raw_type":"Text"},{"id":"pmh:oai:pubmedcentral.nih.gov:10491101","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/10491101","pdf_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC10491101/pdf/nihpp-2023.08.27.554228v1.pdf","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":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"bioRxiv","raw_type":"Text"}],"best_oa_location":{"id":"doi:10.1101/2023.08.27.554228","doi":"https://doi.org/10.1101/2023.08.27.554228","is_oa":true,"landing_page_url":"https://doi.org/10.1101/2023.08.27.554228","pdf_url":"https://www.biorxiv.org/content/biorxiv/early/2023/08/28/2023.08.27.554228.full.pdf","source":{"id":"https://openalex.org/S4306402567","display_name":"bioRxiv (Cold Spring Harbor Laboratory)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2750212522","host_organization_name":"Cold Spring Harbor Laboratory","host_organization_lineage":["https://openalex.org/I2750212522"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"bioRxiv","raw_type":"posted-content"},"sustainable_development_goals":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.9537000060081482}],"sustainable_development_goals_aurora":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.6899999976158142}],"study_designs":[],"awards":[{"id":"https://openalex.org/G2292243094","display_name":"Transgene Persistence in Liver Gene Therapy","funder_award_id":"R01DK048252","funder_id":"https://openalex.org/F4320337357","funder_display_name":"National Institute of Diabetes and Digestive and Kidney Diseases"},{"id":"https://openalex.org/G5684169252","display_name":"Program in Molecular and Cellular Biosciences","funder_award_id":"T32GM071338","funder_id":"https://openalex.org/F4320337354","funder_display_name":"National Institute of General Medical Sciences"},{"id":"https://openalex.org/G6120594151","display_name":"In vivo selection of gene modified hepatocytes","funder_award_id":"R01DK123093","funder_id":"https://openalex.org/F4320337357","funder_display_name":"National Institute of Diabetes and Digestive and Kidney Diseases"},{"id":"https://openalex.org/G9000866549","display_name":"Program in Biomedical Sciences","funder_award_id":"T32GM142619","funder_id":"https://openalex.org/F4320337354","funder_display_name":"National Institute of General Medical Sciences"}],"funders":[{"id":"https://openalex.org/F4320311248","display_name":"National PKU Alliance","ror":"https://ror.org/04622jx20"},{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"},{"id":"https://openalex.org/F4320337354","display_name":"National Institute of General Medical Sciences","ror":"https://ror.org/04q48ey07"},{"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/W4387564437.pdf","grobid_xml":"https://content.openalex.org/works/W4387564437.grobid-xml"},"referenced_works_count":46,"referenced_works":["https://openalex.org/W1568300712","https://openalex.org/W1836427129","https://openalex.org/W1974168476","https://openalex.org/W1979686921","https://openalex.org/W1983670700","https://openalex.org/W1983773316","https://openalex.org/W1984097721","https://openalex.org/W1990591430","https://openalex.org/W2007221068","https://openalex.org/W2021097015","https://openalex.org/W2021718747","https://openalex.org/W2025596234","https://openalex.org/W2039826443","https://openalex.org/W2047821701","https://openalex.org/W2060640171","https://openalex.org/W2068270541","https://openalex.org/W2070536631","https://openalex.org/W2072123484","https://openalex.org/W2083208993","https://openalex.org/W2089582480","https://openalex.org/W2101613203","https://openalex.org/W2104010513","https://openalex.org/W2106070783","https://openalex.org/W2112730584","https://openalex.org/W2125711506","https://openalex.org/W2143096892","https://openalex.org/W2148530915","https://openalex.org/W2461975652","https://openalex.org/W2502200093","https://openalex.org/W2562295703","https://openalex.org/W2603828830","https://openalex.org/W2616215756","https://openalex.org/W2770195502","https://openalex.org/W2903565415","https://openalex.org/W2911604931","https://openalex.org/W2947834998","https://openalex.org/W2998155166","https://openalex.org/W3091313215","https://openalex.org/W3162445048","https://openalex.org/W3170805629","https://openalex.org/W3183349244","https://openalex.org/W4210788876","https://openalex.org/W4292249804","https://openalex.org/W4310640268","https://openalex.org/W6634165798","https://openalex.org/W6665574444"],"related_works":["https://openalex.org/W4403777503","https://openalex.org/W4400135985","https://openalex.org/W3170805629","https://openalex.org/W6939526116","https://openalex.org/W2068270541","https://openalex.org/W1983670700","https://openalex.org/W1995612506","https://openalex.org/W2090195986","https://openalex.org/W1900779987","https://openalex.org/W2014397874"],"abstract_inverted_index":{"BACKGROUND":[0],"AND":[1,116],"AIMS:":[2],"Hepatocyte":[3],"transplantation":[4],"for":[5,75,191],"genetic":[6],"liver":[7,45,97,173],"diseases":[8],"has":[9,23],"several":[10],"potential":[11,189],"advantages":[12],"over":[13],"gene":[14,55],"therapy.":[15],"However,":[16],"the":[17,44,76],"low":[18],"efficiency":[19],"of":[20,43,78,110,184],"cell":[21,113],"engraftment":[22],"limited":[24],"its":[25],"clinical":[26],"implementation.":[27],"This":[28],"problem":[29],"could":[30],"be":[31],"overcome":[32],"by":[33,112],"selectively":[34,59],"expanding":[35],"transplanted":[36,134,185],"donor":[37,122],"cells":[38,85],"until":[39],"they":[40,166],"replace":[41],"enough":[42],"mass":[46],"to":[47,58,80,93,105,128,160],"achieve":[48],"therapeutic":[49,190],"benefit.":[50],"We":[51,179],"previously":[52],"described":[53],"a":[54,81,107,120,188,197],"therapy":[56],"method":[57],"expand":[60,94],"hepatocytes":[61,155,186],"deficient":[62],"in":[63,126,162,170],"cytochrome":[64],"p450":[65],"reductase":[66],"(Cypor)":[67],"using":[68],"acetaminophen":[69],"(APAP).":[70],"Because":[71],"Cypor":[72,130],"is":[73,187],"required":[74],"transformation":[77],"APAP":[79,152],"hepatotoxic":[82],"metabolite,":[83],"Cypor-deficient":[84,154],"are":[86,91],"protected":[87],"from":[88,119,157],"toxicity":[89],"and":[90,132,148,165,196],"able":[92],"following":[95,151],"APAP-induced":[96],"injury.":[98],"Here,":[99],"we":[100],"apply":[101],"this":[102],"selection":[103,139,183],"system":[104],"correct":[106],"mouse":[108],"model":[109],"phenylketonuria":[111,192],"transplantation.":[114],"APPROACH":[115],"RESULTS:":[117],"Hepatocytes":[118],"wild-type":[121],"animal":[123],"were":[124,145],"edited":[125],"vitro":[127],"create":[129],"deficiency":[131],"then":[133],"into":[135],"phenylketonuric":[136],"animals.":[137],"Following":[138],"with":[140,193],"APAP,":[141],"blood":[142,171],"phenylalanine":[143],"concentrations":[144],"fully":[146],"normalized":[147],"remained":[149],"stable":[150],"withdrawal.":[153],"expanded":[156],"<":[158],"1%":[159],"~14%":[161],"corrected":[163],"animals,":[164],"showed":[167],"no":[168],"abnormalities":[169],"chemistries,":[172],"histology,":[174],"or":[175],"drug":[176],"metabolism.":[177],"CONCLUSIONS:":[178],"conclude":[180],"that":[181],"APAP-mediated":[182],"long-term":[194],"efficacy":[195],"favorable":[198],"safety":[199],"profile.":[200]},"counts_by_year":[{"year":2026,"cited_by_count":7},{"year":2025,"cited_by_count":8},{"year":2024,"cited_by_count":3},{"year":2023,"cited_by_count":1}],"updated_date":"2026-10-30T12:39:25.321706","created_date":"2023-10-13T00:00:00"}