ATP7B gene therapy of autologous reprogrammed hepatocytes alleviates copper accumulation in a mouse model of Wilson's disease.

Hongxia CaiXing ChengXiao-Ping Wang

Published in: Hepatology (Baltimore, Md.) (2022)

We established a system of autologous reprogrammed WD hepatocytes and achieved ATP7B gene therapy in vitro. LPC-ATP7B-Heps transplantation demonstrated therapeutic efficacy on copper homeostasis in a mouse model of WD.

Keyphrases

gene therapy
mouse model
cell therapy
bone marrow
liver injury
platelet rich plasma
oxide nanoparticles
drug induced
mesenchymal stem cells