Hepatocellular carcinoma (HCC), also called malignant hepatoma, is one of the deadliest cancers. Through the introduction of a double-stranded RNA to the targeted messenger RNA (mRNA), RNA interference (RNAi) leads to the specific cleavage of the mRNA and efficient silencing of gene expression. Since RNAi could be used to silence genes involved in the development and progression of carcinomas, it has promising therapeutic potential for their treatment. The gene therapy strategy we propose here:
(1) utilize two cancer-specific promoters (one HCC-specific) to open an AND-gated system to target HCC, the selectivity supposed to be extremely high;
(2) is dependent on and hence controllable by a low molecular weight compound;
(3) has the flexibility to be adapted to target any mRNA and, if there are disease-specific promoters, other diseases.
More relevant background information can be seen in Background