The Chemical Biology and Biotherapeutics scientific pillar carries out basic chemical research of fundamental importance to precision medicine, investigating the cellular processes that are impacted by aberrations in gene expression or protein function. Understanding the relationships between gene expression and protein activity is fundamental to our ability to identify and develop patient-specific therapeutics or diagnostic agents.
Focal Challenges
The individual genetic makeup of each patient directly affects the way that proteins (metabolome, glycome, liposome and/or microbiome) are expressed in healthy and diseased states. To fully understand this relationship as the basis for development of patient-specific medicine, we must address three central challenges:
- Examination of large numbers of patient samples to identify gene-driven changes in protein expression that give rise to disease states
- Discovery of novel small molecule therapeutics that can be used to address disease-driven protein expression changes within specific patient cohorts
- Development of precision medicines that can address protein expression changes in specific clinical cohorts with minimal side effects.