HCV感染的基因治疗

Gene Therapy in HCV Infection
Lai Wei

Peking University People’s Hospital, Peking University Hepatology Institute, Beijing, China
On the second day of APSAL, Professor Lai Wei presented a report on gene therapy in hepatitis C virus infection. He introduced some potential new target for hepatitis C gene therapy, like IRES in the in the 5' NCR of HCV, hnRNP D , miR-122, as well as a few methods in hepatitis C gene therapy, such as ribozymes, antisense oligodeoxynucleotides, and RNA interference (RNAi). Several ribozymes targeting highly conserved HCV sequences were found to effectively degrade the HCV RNA strands in vitro. In vitro study, and study in BALB/c mice, showed effective inhibition on HCV mRNA expression by antisense oligonucleotides complementary to the HCV 5'NCR. Using the characteristic of RNAi targeting viral mRNA for degradation by cellular enzymes, RNAi might imply a potential therapic strategy. Synthetic siRNA specific for HCV 5’NCR were designed and observed for inhibition efficacy on HCV.

   在APASL会议的第二天，魏来教授做了关于HCV感染基因治疗的大会报告。他介绍了一些新近发现的基因治疗潜在靶点，如位于HCV 5' 非翻译区（NCR）的IRES，以及一些相关基因治疗方法，如核酶、反义寡核苷酸连、RNA干扰（RNAi），它们已经体外及动物试验证明可有效抑制HCV复制及表达。