Transplantation of genetically engineered hepatocytes

摘要

Hepatocyte transplantation and liver-directed gene therapy are being developed in parallel. Hepatocytes could serve as vehicles for ex-vivo gene therapy, as demonstrated in the first long-term liver-directed gene therapy in low-density lipoprotein (LDL) receptor-deficient Watanabe heritable hyperlipidaemic rabbits1. On the other hand, gene transfer could be used to immortalize the cells2'3, augment the expression of specific genes4 to induce massive repopulation of the liver5 and to prevent immune rejection.