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

A microRNA is a short non-coding RNA that has the ability to target mRNA, to mediate its degradation and to regulate gene expression at post-transcriptional level. This post-transcriptional gene silencing (also known as RNA interference) proved widely applicable in gene therapy, albeit with undesirable effects due to low specificity. Mature miRNA is processed form the primary transcript pri-miRNA, therefore an “AND” gate system could be built based on specific structural properties that define pri-miRNAs, in order to improve such specificity. We then also managed to engineer disease-specific promoters and RNA-dependent RNA polymerase to process the logic information. CPU_CHINA focused on hepatocellular carcinoma (HCC) as the disease-therapeutic setting to be applied on with this strategy. But the concept definitely possesses the flexibility that can be discussed over other disease settings and the corresponding necessary gene therapy, which supports a New Application of Genetically Engineered Machine.