Computational approaches in design of nucleic acid-based therapeutics

Abstract

Recent advances in computational and experimental methods have led to novel avenues for therapeutic development. Utilization of nucleic acids as therapeutic agents and/or targets has been recently gaining attention due to their potential as high-affinity, selective molecular building blocks for various therapies. Notably, development of computational algorithms for predicting accessible RNA binding sites, identifying therapeutic target sequences, modeling delivery into tissues, and designing binding aptamers have enhanced therapeutic potential for this new drug category. Here, we review trends in drug development within the pharmaceutical industry and ways by which nucleic acid-based drugs have arisen as effective therapeutic candidates. In particular, we focus on computational and experimental approaches to nucleic acid-based drug design, commenting on challenges and outlooks for future applications.