The focus group “Gene-regulatory Mechanisms” consists of Hans Fischer Senior Fellow Prof. Lothar Hennighausen (NIH/NIDDK), his host Prof. Bernhard Küster (TUM School of Life Sciences) and Dr. Markus List (TUM School of Life Sciences).
High-precision genome editing tools have the potential to cure human genetic diseases. The most important question now is to learn which gene edits can lead to a cure without risking severe side effects. Multifactorial diseases are often caused by dysregulation of genes rather than dysfunction. It is thus not sufficient to simply inactivate or repair dysfunctional genes. Instead, we need to elucidate and understand how genes are regulated to allow for more fine-grained interventions that restore the gene-regulatory balance found in healthy cells. This is a daunting task as multiple of the more than 20,000 genes in a mammalian genome may be affected in a disease, each of which is controlled by several regulatory elements. While the regulation of individual genes has been well studied, we lack a reliable way to predict the presence of genuine biologically relevant regulatory elements. In this focus group we seek to use machine learning and artificial intelligence to develop computational tools and methods that offer unprecedented insights into the underlying principles of gene regulation, which will eventually allow more targeted genome-editing studies aimed at curing human diseases.