Review of the research and development status of siRNA drugs for gene therapy

Abstract

Gene therapy is a new kind of therapy technology, which can modify or manipulate gene expression, gene silencing, and other functions, or change the biological characteristics of cells, so as to achieve the goal of curing disease at the gene level. It is not restricted by the drug-forming property of the target protein and has obvious advantages compared with traditional protein drug therapy. To develop small interfering ribonucleic acid (siRNA) drugs for gene therapy, the mechanism of ribonucleic acid interference (RNAi), which was discovered more than 30 years ago, can be taken as a basis. Later, scientists' unremitting exploration made siRNA drugs develop rapidly. So far, there are five kinds of siRNA drugs listed, with good application prospects. This paper aims to summarize the existing siRNA drugs and explore their shortcomings, as well as some existing improvement measures, thereby pointing out the direction of drug development and providing some help for the better application of siRNA drugs. According to the analysis, it is found that there are still some dilemmas in the research and development of siRNA drugs in terms of stability, targeting accuracy, long-term effectiveness, efficiency, and safety. Meanwhile, the performance of the drug delivery system also determines whether the drug can play an effective role. Some improvements have been proposed in this paper, such as modifying drug molecules or improving delivery systems to help drugs function better, which could be the direction of future drug progress.