Therapeutic liver cell transplantation to treat a genetic liver defect
Melanie Willimann, Hiu Man Grisch‐Chan, Nicole Rimann, Tanja Rothgangl, Martina Hruzova, Gerald Schwank, 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.
bioRxiv · Jun 26, 2024 · PreprintMolecular Biology