Liver-directed gene therapy

Spectacular advances in vectorology, modulation of gene expression, and gene editing have led to a resurgence of interest in gene therapy both for interrogating the mechanisms of biological processes and for treatment of diseases. Liver-directed gene therapy can be used for diverse therapeutic strategies. Targets of liver-directed gene therapy include inherited metabolic disorders, and also acquired conditions, such as infectious and neoplastic diseases, cirrhosis of the liver, and immune rejection of transplants. Gene transfer into isolated hepatocytes can be used as an adjunct to cell transplantation for ex vivo gene therapy, for inducing preferential proliferation of the transplanted hepatocytes, or for preventing immune rejection. The chapter lists some of the inherited disorders that are targets of gene therapy. Oncolytic viruses that preferentially replicate in tumor cells, have expanded the role of cancer gene therapy as both tumoricidal and immune adjuvant therapy.