The road toward microRNA therapeutics

Within the last 15 years, microRNAs (miRNAs) have emerged as powerful regulators of gene expression. Highly conserved, these small RNAs appear to regulate nearly every aspect of eukaryotic biology. Perturbation of miRNA expression has been correlated with cancer and, more recently, a variety of diseases; however, the more striking observation is that manipulation of miRNA levels can control disease phenotypes. Hence, the race to bring the first miRNA therapeutic to the market has begun. This review provides an overview of the history and mechanism of miRNAs, the ongoing efforts to develop miRNA therapies, and the nucleic acid technologies that may be key to the success of a miRNA therapeutic.