Drug target miRNAs: chances and challenges

• miRNAs are high-potential drug targets.
• Development of miRNA-targeting drugs is challenging due to their chemical structure comprising short ribonucleic acids.
• Anti-sense agents targeting miRNAs have been successful when used in advanced formulations.
• Small-molecule modulators that bind to RNAi-involved proteins have good chances to become a new drug class.

miRNAs, short ribonucleic acid gene regulators, are increasingly popular drug targets. Traditionally ‘undruggable’ proteins can be targeted via their miRNA gene regulators, enabling the treatment of diseases that, at present, seem impossible to cure. However, addressing miRNAs requires innovation at the level of drug discovery. This review article outlines the potential of miRNAs as drug targets, focuses on the challenges of developing miRNA-targeting drugs, and surveys new advances. The aim is to provide an orientation guide for scientists, as well business analysts, to help them navigate the jungle of different approaches in miRNA drug discovery.