{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Cai H"],"funding":["National Natural Science Foundation of China"],"pagination":["1046-1057"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC9790736"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["76(4)"],"pubmed_abstract":["<h4>Background and aims</h4>Wilson's disease (WD) is a rare hereditary disorder due to ATP7B gene mutation, causing pathologic copper storage mainly in the liver and neurological systems. Hepatocyte transplantation showed therapeutic potential; however, this strategy is often hindered by a shortage of quality donor cells and by allogeneic immune rejection. In this study, we aimed to evaluate the function and efficacy of autologous reprogrammed, ATP7B gene-restored hepatocytes using a mouse model of WD.<h4>Approach and results</h4>Sufficient liver progenitor cells (LPCs) were harvested by reprogramming hepatocytes from ATP7B<sup>-/-</sup> mice with small molecules, which exhibited strong proliferation and hepatic differentiation capacity in vitro. After lentivirus-mediated mini ATP7B gene t"],"journal":["Hepatology (Baltimore, Md.)"],"pubmed_title":["ATP7B gene therapy of autologous reprogrammed hepatocytes alleviates copper accumulation in a mouse model of Wilson's disease."],"pmcid":["PMC9790736"],"funding_grant_id":["81671103"],"pubmed_authors":["Wang XP","Cheng X","Cai H"],"additional_accession":[]},"is_claimable":false,"name":"ATP7B gene therapy of autologous reprogrammed hepatocytes alleviates copper accumulation in a mouse model of Wilson's disease.","description":"<h4>Background and aims</h4>Wilson's disease (WD) is a rare hereditary disorder due to ATP7B gene mutation, causing pathologic copper storage mainly in the liver and neurological systems. Hepatocyte transplantation showed therapeutic potential; however, this strategy is often hindered by a shortage of quality donor cells and by allogeneic immune rejection. In this study, we aimed to evaluate the function and efficacy of autologous reprogrammed, ATP7B gene-restored hepatocytes using a mouse model of WD.<h4>Approach and results</h4>Sufficient liver progenitor cells (LPCs) were harvested by reprogramming hepatocytes from ATP7B<sup>-/-</sup> mice with small molecules, which exhibited strong proliferation and hepatic differentiation capacity in vitro. After lentivirus-mediated mini ATP7B gene t","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Oct","modification":"2026-07-14T16:00:33.148Z","creation":"2025-04-05T08:47:27.354Z"},"accession":"S-EPMC9790736","cross_references":{"pubmed":["35340061"],"doi":["10.1002/hep.32484"]}}