Therapeutic applications of noncoding RNAs.

Abstract

In this review, we summarize the basic principles underlying the therapeutic use of nonprotein coding (nc)RNAs, such as microRNA (miRNA) and long noncoding RNA, in the cardiovascular field, focusing, where possible, on recent advances that may lead to translation to the clinic for heart failure. The number of individual miRNAs associated with a given aspect of heart disease is increasing rapidly, as is the data on miRNA profiles in normal and diseased myocardium. Less is known on the role of long noncoding RNA, and to date only a few have been studied in the heart. Novel oligonucleotide-based therapies have started to trickle into the clinic, but strategies focusing on ncRNA are still in a clinical/preclinical trial phase. The discovery of functional ncRNAs is leading to a better understanding of the mechanisms underlying cardiovascular physiology. Dysregulation of ncRNAs is being increasingly associated with many diseases affecting the heart and in certain instances may have a pathogenic role. Therapeutic intervention aimed at opposing ncRNA misexpression has been widely demonstrated to be effective in blunting disease in animal models, and may thus have potential in the clinical setting.