Approved research

Combining genetic analysis of disease related phenotypes with in vitro disease models for development of high efficacy and low toxicity therapeutics

Lay summary

The development of new therapeutics for human diseases is a challenging process with high costs and failure rates. A major cause of these failures is the lack of good pre-clinical in vitro models for many diseases. Recent studies show that investigating the genetic and environmental influences on diseases can guide the drug discovery process by identifying key driver genes, biological processes and cell types. Therefore, we will use the UK Biobank resource to implement and develop new statistical and machine learning approaches for such analysis, with the aim of guiding in vitro disease model development, and identifying and validating new therapeutic targets. We aim to first apply this approach to develop therapeutics for neurological, metabolic and immunological disorders. Considering that our project involves the generation of hypotheses as well as experimental validation of new therapeutics, we expect it to take several years. Nevertheless, a successful outcome of this project can have a significant effect on our ability to develop new, effective therapeutics, and hence on the well-being of many patients.