The RNA Revolution and RNA-Based Therapeutics: The Good, the Bad, and the Ugly

RNA based medicines have been leading the way in what has been called a 'golden age of medicine.' The covid-19 mRNA vaccines, developed in 2020, represented the first of these RNA-based medicines. Since then, additional therapies using RNA have been developed which will open the doors for a potential renaissance of therapeutics. In this course, we will discuss the science behind this innovative platform as well as describe recent applications of RNA-based therapeutics in virology and cancer. We will provide the essential background on the revolution in RNA biology (what RNA is, how it is made, how its structure is determined, and its functions in the cell) and describe how the structure of RNA is important to its function and in the design and development of novel RNA-based therapeutics. We will also discuss how technological advances have optimized mRNA-based vaccine stability and improved delivery methods for therapeutics. In addition, we will provide an overview of the effectiveness of these therapies in mounting a robust immune response, describing immune cell subsets that have not been widely reported in the media but which likely played central roles in protection against disease. Finally, we will look to the future by describing next generation RNA-based therapies that, while not yet approved by the FDA, have had encouraging results from early clinical trials. The class will be a combination of lecture and discussion, and will be suitable for people who do not necessarily have an in-depth background in science, as findings from peer-reviewed literature will be translated into easily understandable language.