LIVER-DIRECTED GENE-TRANSFER AND APPLICATION TO THERAPY

The liver is an important and attractive target for the development of gene therapy strategies. Many genetic diseases are manifested in the liver, and both infectious and malignant diseases affect this organ. R etroviral and adenoviral vectors have been shown to infect hepatocytes with varying efficiently in vitro and in vivo. The presence of unique receptors at the cellular membrane of hepatocytes has stimulated the development of transfer strategies based on receptor targeting of vect ors. The results of a first clinical trial for gene therapy in the liv er based on ex vivo gene delivery has shown both the feasibility and t he limits of current technology. This review discusses both existing v ectors and strategies and prospective developments towards liver-direc ted gene therapy of genetic and malignant diseases.