Therapeutic advances in MicroRNA targeting.

ABSTRACT

In the last 10 years it has become increasingly clear that a large class of small noncoding RNAs, known as microRNAs (miRNAs) are potent and crucial regulators of important cellular processes such as differentiation, growth, and survival. miRNAs regulate gene expression through binding to 3' UTRs of target messenger RNAs whereby inducing either messenger RNA degradation or inhibition of protein translation. Although we have only just begun to gain some insight into the biology surrounding miRNAs, their apparent relevance and potency during the onset and progression of disease has generated a lot of interest in assessing the feasibility of therapeutic regulation of miRNAs. As a result of the short RNA nature of miRNAs and lessons learned from small interfering RNA therapeutics and gene therapy, within a timespan of a few years, incredible progress has been made in advancing miRNA regulation into the clinic. We summarize the various therapeutic tools that are currently being investigated to manipulate miRNAs with a special focus on cardiovascular disease and speculate on the future developments of miRNA therapeutics.