Pre-clinical trials using hepatic gene delivery.
The development of gene therapy as a potential technique for treating serious metabolic or infectious disorders has generated much interest. The general applicability of gene therapy depends on the efficient transfer of the desired gene to specific tissues and cells. One of the most attractive sites for gene transfer is the liver because it plays a major role in many metabolic processes and is involved in a large variety of diseases. Nonviral strategies have been conceived for delivering genes to the liver but this approach is still at the preclinical stage. This review outlines the more commonly used approaches and discusses the progress that has been made toward developing a widely applicable, clinically relevant gene transfer procedure for the liver.