MicroRNAs as therapeutic targets in human diseases.

MicroRNAs (miRNAs) are important endogenous regulators of gene expression. The specific regulation at both the transcription and the translation level (inhibition or mRNA degradation) opens an avenue to use these small RNA molecules as potential targets for the development of novel drugs as well as for the diagnosis of several human diseases. Important information about the role of a miRNA in disease can be deduced by mimicking or inhibiting its activity and examining its impact on the phenotype/behaviour of the cell or organism. Modulating the activity of a miRNA is expected to lead to improvement in disease symptoms and this implies that the target miRNA plays an important role in the disease. It is also now possible to develop miRNA-based therapeutic products that can either increase or decrease the levels of proteins in pathophysiological conditions such as cancer, cardiovascular diseases, viral diseases, metabolic disorders and programmed cell death. The commercial potential of miRNA and related drugs is expected to exponentially increase within the next few years, yet there are several areas in miRNA biology and delivery that need to be extensively investigated.