Experimental Therapeutics: Antisense and Gene Therapy Cardiovascular Drugs

Abstract

Exploiting the Watson: Crick base recognition of ribonucleic acid for the discovery and development of antisense therapeutics expands treatment opportunities in cardiovascular medicine. The chemical approaches to synthetic oligomers with drug-like properties have matured but several distinct strategies are possible. However, an expanding role for a variety of newly described RNA species will provide an even greater flexibility in design of new antisense drugs. A review of antisense agents in development are described for vascular disease, hyperlipemia, diabetes, myocardial ischemia, and heart failure is provided. The recent FDA approval of Kynamro creates enthusiasm to develop even more new antisense drugs but the presence of anti-antisense antibodies suggests caution and a need to continue to explore more refined approaches to antisense technology.