A gene therapy strategy to target hepatocellular carcinoma based on conditional RNA interference

MicroRNA(miRNA) is a short non-coding RNA, which has the ability to target mRNA and medicate its degradation to downgrade gene expression at transcription level. This mediation effect is called post-transcriptional gene silencing (PTGS, which is also know as RNA interference). RNA interference(RNAi) has proved its potential as gene therapy. The mature miRNA are cleaved from pri-miRNA which gives us possibility to make it conditional. An “AND” gate system based on specific promoters and RNA-dependent RNA polymerase(RdRp) was designed to avoid undesirable effects due to low selectivity. Together, they formed our conditional RNA gene therapy. This year, CPU_CHINA is focusing on hepatocellular carcinoma (HCC). But significantly, the promoters and the target here can be replaced with other disease-specific promoters and target, in gene therapy for the corresponding diseases which enables our project to be a remarkable novel application.