Title: Advancing statistical genetics tools for reliable drug target discovery and treatment optimization
This program aims to develop and apply advanced statistical genetics tools to address key methodological challenges in identifying the potential causal effects of circulating molecules on complex diseases. By strengthening causal inference, these innovations will accelerate the discovery of novel drug targets, optimize therapeutic regimens, and enable more precise treatment strategies to improve health outcomes.
From a methodological perspective, the program will enhance the robustness of Mendelian randomization, a genetics-based approach for causal inference, to ensure more reliable results across populations. It will develop new frameworks for drug target discovery that account for pre-existing health conditions, generating actionable insights for more tailored therapies. The program will also refine non-linear analytical approaches to address known sources of bias, enabling more accurate and clinically meaningful dose–response evaluations.
Building on these methodological advances, the program will pursue transformative applications. It will conduct systematic drug target discovery by integrating multi-omics data and genetic resources from populations representing diverse genetic ancestries, as well as individuals with varying combinations of pre-existing conditions. The program will characterize dose-dependent effects of identified targets to inform optimal therapeutic dosing. In addition, it will evaluate combined therapies, identify potential synergistic effects, and support the development of precision treatment strategies.
By integrating theoretical innovation with large-scale applied research, this program seeks to establish a robust and generalizable framework for drug target discovery and treatment optimization. These efforts will help bridge the gap between genetic discovery and clinical translation, advancing personalized medicine and improving health outcomes for populations in the United States and worldwide.