Pharmacological potential of RNAi--focus on miRNA.
ABSTRACT RNA interference (RNAi) is a cellular process that is widely used as a research tool to control the expression of specific genes and has the potential as a therapeutic strategy for many diseases. MicroRNAs (miRNAs) and short interfering RNAs (siRNAs) are the two principal categories of small RNAs that induce RNAi in a broad spectrum of eukaryotic organisms including human cells. miRNAs have an enormous capacity to regulate multiple genes and the expression of approximately 30% of the human genes is affected by these non-coding RNAs. Because many miRNAs are specifically expressed during disease, miRNAs are interesting tools for pharmacology and understanding the function of specific miRNAs will help to identify novel drug targets. Furthermore, miRNA-based diagnostics as well as therapeutic interventions are being developed for clinical applications.