Recent advances in liver-directed gene transfer vectors

Abstract

Background: Gene therapy as part of modern molecular medicine holds great promise for the treatment of hepatocellular carcinoma (HCC) and has the potential to bring a revolutionary era to cancer treatment. For the past decade various viral and non-viral vectors have been engineered for improved liver gene therapy.

Data resources: An English-language literature search using MEDLINE (2004), Index Medicus (2004) and bibliographic reviews of books and review articles. Liver-directed gene transfer vectors and their history and recent clinical applications.

Results: The ultimate goal of liver-directed gene therapy for HCC is the stable expression of a therapeutic transgene in a significant proportion of hepatocytes. The design of a vector system providing efficient and stable gene engraftment and expression in human hepatocytes is still a challenging issue. The advantages and disadvantages of the genetically engineered vector of viral or non-viral origin are discussed with respect to their essential relevance.

Conclusion: Liver gene therapy has a long way to go and efficient and innocuous liver-directed gene transfer vectors are therefore urgently required.