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Recent advances in liver-directed gene transfer vectors |
Dong Xia, Ming-Man Zhang and Lu-Nan Yan |
Chengdu, China
Author Affiliations: Department of General Surgery, West China Hospital, Sichuan University, Chengdu 610041, China (Xia D, Zhang MM and Yan LN )
Corresponding Author: Lu-Nan Yan, MD, Department of General Surgery, West China Hospital, Sichuan University, Chengdu 610041, China (Tel: 86-28-85422477; Fax: 86-28-85423724; Email: YanLu-nan@hotmail.com) |
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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.
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