Liver gene therapy: prospects in the new century

ABSTRACT

Novel and exciting techniques have been developed for the genetic modification of hepatocytes. There are five broadly defined indications for direct gene transfer in liver therapy 1] Gene replacement therapy, 2] Gene expression therapy, 3] Viral enzyme prodrug therapy, 4] Inhibition of gene expression, and 5] Repair of abnormal genes. The two main methods of gene delivery to the liver are 1] viral vectors including Retroviruses, Adenoviruses, Adeno-associated, Simian virus 40, hybrid virus vectors, and 2] non-viral methods involving attachment of a therapeutic gene to a carrier. These may be either polymer based cationic carriers [conjugates] or lipid based vectors [liposomes]. The last decade of the second millennium has seen a lot of research work done in the field of genetics. Based on the many advances in the field, we hope that there is bright future for clinical applications of gene therapy for hepatic diseases