Targeting MicroRNAs With Small Molecules: From Dream to Reality

Abstract

Currently, one out of four deaths in the United States is due to cancer; therefore, every major therapeutic advance in cancer has the potential to save many lives. One of the most fascinating discoveries in the area of molecular oncology in the past decade is that, in cancer, alterations in both protein-coding genes and noncoding RNAs, known as microRNAs (miRNAs), complement each other. In this article, we focus on a novel idea involving the use of small molecules to target overexpressed miRNAs as a new therapy for diseases, particularly cancer, in humans. Clinical Pharmacology & Therapeutics (2010) 87 6, 754–758. doi: 10.1038/clpt.2010.46