Xia Dong, Zhang Ming-Man, Yan Lu-Nan
Department of General Surgery, West China Hospital, Sichuan University, Chengdu 610041, China.
Hepatobiliary Pancreat Dis Int. 2004 Aug;3(3):332-6.
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.
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.
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.
Liver gene therapy has a long way to go and efficient and innocuous liver-directed gene transfer vectors are therefore urgently required.
基因治疗作为现代分子医学的一部分，在肝细胞癌（HCC）治疗方面前景广阔，有望为癌症治疗带来一个变革性的时代。在过去十年中，人们设计了各种病毒和非病毒载体以改进肝脏基因治疗。
使用MEDLINE（2004年）、《医学索引》（2004年）进行英文文献检索，并查阅书籍和综述文章的文献综述。肝脏定向基因转移载体及其历史和近期临床应用。
肝癌肝脏定向基因治疗的最终目标是使治疗性转基因在相当比例的肝细胞中稳定表达。设计一种能在人肝细胞中提供高效稳定基因植入和表达的载体系统仍然是一个具有挑战性的问题。讨论了病毒或非病毒来源的基因工程载体的优缺点及其本质相关性。
肝脏基因治疗还有很长的路要走，因此迫切需要高效且无害的肝脏定向基因转移载体。
