Molecular BiologybioRxivPreprint
Therapeutic liver cell transplantation to treat a genetic liver defect
Melanie Willimann, Hiu Man Grisch‐Chan, Nicole Rimann…·Beat Thöny
This paper describes a gene therapy approach using liver cell transplantation to treat PKU in mice by modifying hepatocytes to resist paracetamol and replace diseased cells.
DOI 10.1101/2024.06.26.600783