RNA Biology and Therapeutics

RNA molecules can store and transfer genetic information, as well as regulate cellular processes through enzymatic activity and their interaction with other biomolecules. In the past decade, advances in next-generation sequencing have revealed new classes of RNAs and the multiple layers of information and functions they carry. The newly discovered functions of different classes of RNA molecules and their roles in human development and disease have led to the emergence of RNA therapeutics. This course explores the intersection of modern basic and translational research on RNA biology along with the biotechnology industry's drug development efforts around RNA therapeutics. The course offers a unique opportunity for students, researchers, and biotechnology innovators to expand their knowledge about the growing science of RNA therapeutics and to develop a deep understanding of RNA-focused drug development in the biotech industry. This course opens with an introduction to the RNA world, including evolutionary theories about biomolecules, fundamental concepts related to the structure and functions of RNAs, current classifications of RNA molecules, and modern tools and techniques for studying RNAs. The course then covers current classes of RNA molecules and their roles in normal biology and in disease. This course focuses on a number of classes of RNA therapeutics including drugs that target normal or abnormal RNA transcripts and drugs and vaccines composed of RNAs.