Ribonucleic acid (RNA) biology has emerged as one of the most influential areas in modern biology and biomedicine. The discovery of numerous new classes of RNAs and their functions in a wide spectrum of biological processes has revolutionized molecular biology, with profound implications for clinical sciences. Key areas of current research within the Intramural Research Program (IRP) include the elucidation of RNA biogenesis pathways, the determination of RNA structures, the identification of functions for the various classes of RNAs, establishing the role of RNA in disease, and the exploration of RNA-based- and RNA-targeted therapies.
The IRP has taken a leadership role in the development of a comprehensive program for the investigation and therapeutic exploitation of RNA, including antisense therapy, RNA interference, and RNA silencing. The goal is to capitalize on the IRP’s solid foundation in genetics and molecular biology to understand the role of RNA biology in health and disease.
Sub-divisions of active IRP research into RNA biology and therapeutics include:
- Pre-mRNA processing, such as capping, splicing, and polyadenylation
- Biogenesis and functions of noncoding RNAs
- Subcellular trafficking and localization
- RNA folding
- RNA structure
- Modifications including editing
- RNA-protein interactions
- RNA-RNA interactions including microRNAs
- RNA stability
- RNA viruses
- Transcriptional regulation
The IRP already has contributed to promising antisense therapies for hemorrhagic fever viruses, amyotrophic lateral sclerosis, and numerous cancers. The IRP’s RNAi screening facility provides the tools to screen whole genomes and pathways on an industrial scale, and intramural researchers have identified a multitude of genes that control critical processes and pathways in cancer and neurodegenerative diseases.