RNA interference (RNAi) is a convenient and useful gene suppression technology induced by small interfering RNA (siRNA) composed of 21-nucleotide long double-stranded RNA. The successful application of RNAi for clinical use is expected for a long time. Although siRNA drug is categorized into a nucleic acid drug, it has a prominent advantage that genetic function can be suppressed by destroying mRNA at the posttranscriptional level without wounding genomic DNA. Nevertheless, unfortunately there are no siRNA certified as pharmaceuticals passing through clinical trials, since there are several problems, such as gene suppression efficiency, stability in blood stream, or other undesirable effects. Here, we describe the current status and future prospects for clinical application of the siRNA nucleic acid drug.
Part of the book: Gene Expression and Regulation in Mammalian Cells