{"doi":"10.1126/scitranslmed.abg3047","title":"Therapeutic liver repopulation by transient acetaminophen selection of gene-modified hepatocytes","abstract":"shRNA were administered to neonatal mice. Hepatocytes lacking the essential cofactor of Cyp enzymes, NADPH-cytochrome p450 reductase (Cypor), were selected in vivo by acetaminophen administration, replacing up to 50% of the hepatic mass. Acetaminophen treatment of the mice resulted in over 30-fold expansion of transgene-bearing hepatocytes and achieved therapeutic thresholds in hemophilia B and phenylketonuria. We conclude that therapeutically modified hepatocytes can be selected safely and efficiently in preclinical models with a transient regimen of moderately hepatotoxic acetaminophen.","journal":"Science Translational Medicine","year":2021,"id":168633,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":30,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9527,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2021-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":632127,"name":"Amita Tiyaboonchai","orcid":"0000-0002-0116-7393","position":1,"is_corresponding":false},{"id":698114,"name":"Sean Nygaard","orcid":"0000-0002-8239-6584","position":2,"is_corresponding":false},{"id":279342,"name":"Jeffrey Posey","orcid":"0000-0001-5205-8261","position":3,"is_corresponding":false},{"id":639736,"name":"A. Mack Peters","orcid":null,"position":4,"is_corresponding":false},{"id":440064,"name":"Shelley R. Winn","orcid":"0000-0002-3035-0584","position":5,"is_corresponding":false},{"id":598827,"name":"Alessio Cantore","orcid":"0000-0002-9741-997X","position":6,"is_corresponding":false},{"id":598833,"name":"Luigi Naldini","orcid":"0000-0002-7835-527X","position":7,"is_corresponding":false},{"id":403794,"name":"Cary O. Harding","orcid":"0000-0001-9146-3112","position":8,"is_corresponding":false},{"id":279344,"name":"Markus Grompe","orcid":"0000-0002-6616-4345","position":9,"is_corresponding":false},{"id":698113,"name":"Anne Vonada","orcid":"0000-0003-0398-1898","position":0,"is_corresponding":true}],"reference_count":34,"raw_metadata":null,"created_at":"2026-07-18T23:46:06.853307Z","pmid":"34108249","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}