RNA therapeutics

  1. Adrian R. Krainer2
  1. 1Rosalind Franklin University of Medicine and Science, Chicago, Illinois 60064, USA
  2. 2Cold Spring Harbor Laboratory, Cold Spring Harbor, New York 11724, USA
  1. Corresponding author: krainer{at}cshl.edu

Abstract

“RNA therapeutics” refers to a disease treatment or drug that utilizes RNA as a component. In this context, RNA may be the direct target of a small-molecule drug or RNA itself may be the drug, designed to bind to a protein, or to mimic or target another RNA. RNA has gained attention in the drug-development world, as recent clinical successes and breakthrough technologies have revolutionized the drug-like qualities of the molecule or its usefulness as a drug target. In this special issue of RNA, we gathered expert perspectives on the past, present, and future of the field, to serve as a primer and also a challenge to the broad scientific community to incorporate RNA into their experimental design and problem-solving process, and to imagine and realize the potential of RNA as a therapeutic drug or target.

This article, published in RNA, is available under a Creative Commons License (Attribution-NonCommercial 4.0 International), as described at http://creativecommons.org/licenses/by-nc/4.0/.

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