ABSTRACT

RNA therapeutics that use mRNA to encode vaccines and other therapeutic proteins are just one example of how RNA can be used to prevent and treat disease. Here I describe newer classes of therapeutics that are also just reaching the clinic. I describe how all the insights from flipons and RNA editing combine to offer new therapeutic approaches to a wide range of diseases. I summarize existing RNA therapeutics that have solved many problems in delivery, stability, and specificity of RNA-based drugs. The applications based on RNA editing rival the proposed applications for CRISPR DNA without the risks of irreversibly modifying the genome.