摘要
BACKGROUND: Gene therapy as part of modern molecu-lar medicine holds great promise for the treatment of hepa-tocellular carcinoma (HCC) and has the potential to bringa revolutionary era to cancer treatment. For the past de-cade various viral and non-viral vectors have been engi-neered for improved liver gene therapy.DATA RESOURCES: An English-language literature searchusing MEDLINE (2004), Index Medicus (2004) and biblio-graphic reviews of books and review articles. Liver-directedgene transfer vectors and their history and recent clinicalapplications.RESULTS: The ultimate goal of liver-directed gene therapyfor HCC is the stable expression of a therapeutic transgenein a significant proportion of hepatocytes. The design of avector system providing efficient and stable gene engraft-ment and expression in human hepatocytes is still a chal-lenging issue. The advantages and disadvantages of the ge-netically engineered vector of viral or non-viral origin arediscussed with respect to their essential relevance.CONCLUSION: Liver gene therapy has a long way to goand efficient and innocuous liver-directed gene transfer vec-tors are therefore urgently required.
BACKGROUND: Gene therapy as part of modern molecu-lar medicine holds great promise for the treatment of hepa-tocellular carcinoma (HCC) and has the potential to bringa revolutionary era to cancer treatment. For the past de-cade various viral and non-viral vectors have been engi-neered for improved liver gene therapy.DATA RESOURCES: An English-language literature searchusing MEDLINE (2004), Index Medicus (2004) and biblio-graphic reviews of books and review articles. Liver-directedgene transfer vectors and their history and recent clinicalapplications.RESULTS: The ultimate goal of liver-directed gene therapyfor HCC is the stable expression of a therapeutic transgenein a significant proportion of hepatocytes. The design of avector system providing efficient and stable gene engraft-ment and expression in human hepatocytes is still a chal-lenging issue. The advantages and disadvantages of the ge-netically engineered vector of viral or non-viral origin arediscussed with respect to their essential relevance.CONCLUSION: Liver gene therapy has a long way to goand efficient and innocuous liver-directed gene transfer vec-tors are therefore urgently required.
基金
This study was supported by a grant from the National Natural Science Foun-dation of China (No. 30170925).
