With the many overlapping and redundant molecular pathways in cells, it is crucial to identify and rigorously validate the targets underlying disease progression. NCATS experts develop new methods, approaches, tools and technologies to validate targets in a more efficient and predictable fashion, leading to the creation of safe and effective products. This process greatly increases the likelihood that researchers will be able to develop robust, specific assays to facilitate drug discovery.
RNA-based technologies can provide insights into the molecular targets underlying a wide variety of diseases, and these discoveries can lead to the development of novel therapies. Scientists in NCATS’ RNA interference (RNAi) program use gene silencing to better understand gene function. These researchers employ high-throughput RNAi screens to illuminate a range of biological processes, such as which genes that affect the activity of therapeutic agents and novel components of signaling pathways. Investigators from other NIH Institutes and Centers collaborate with NCATS’ RNAi experts to develop assays, conduct genome-wide siRNA screens, and perform rigorous informatics and follow-up.
The Extracellular RNA (ExRNA) Communication program, which provides funding to outside researchers on a competitive basis, aims to explore the basic biology of exRNAs to develop better biomarkers, which could improve understanding and diagnosis of many different diseases.