It is now appreciated that over 90% of the human genome is comprised of noncoding RNAs that have the ability to affect other components of the genome and regulate gene expression. This has galvanized the development of RNA-based therapeutics for a myriad of diseases, including cancer, inflammatory conditions, and cardiovascular disease. Several classes of RNA therapeutics are currently under clinical development, including antisense oligonucleotides, small interfering RNA, and microRNA mimetics and inhibitors. The field of antisense technology saw a huge leap forward with the recent Food and Drug Administration approval of the first antisense therapy, directed against apolipoprotein B, for the treatment of familial hypercholesterolemia. In addition, recent progress in the development of approaches to inhibit microRNAs has helped to illuminate their roles in repressing gene networks and also revealed their potential as therapeutic targets. In this review, these exciting opportunities in the field of drug discovery, with a focus on emerging therapeutics in the field of cardiovascular disease, are summarized.
Marc and Ruti Bell Vascular Biology and Disease Program, Department of Cardiology, New York University School of Medicine, New York, NY.
Reprints: Kathryn J. Moore, PhD, Marc and Ruti Bell Vascular Biology and Disease Program, New York University School of Medicine, New York, NY 10016 (e-mail: firstname.lastname@example.org).
Research on microRNAs in the Moore Laboratory is supported by the NIH (R01HL108182), and E.J.H. is supported by an NIH Training Grant (NIH/NHLBI grant T32HL098129). K.J.M. is a member of the miR-33 Clinical Advisory Board of Regulus Therapeutics, a microRNA therapeutics company.
The authors report no conflicts of interest.
Received March 29, 2013
Accepted May 22, 2013