Therapeutic Advances in microRNA Targeting

Montgomery RL, van Rooij E.

miRagen Therapeutics, 6200 Lookout Rd, Boulder, CO 80301, USA.

In the last ten years it has become increasingly clear that a large class of small non-coding 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 mRNAs whereby inducing either mRNA degradation or inhibition of protein translation. While we have only just began to gain some insights 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. Due to the short RNA nature of miRNAs and lessons learned from siRNA therapeutics and gene therapy, within a timespan of a few years incredible progress has been made in advancing miRNA regulation into the clinic. Here, 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.

http://www.ncbi.nlm.nih.gov/pubmed/20729755