Defining novel resilience pathways in rare monogenic disorders
Daniel Petersheim - Hector Fellow Christoph Klein
In the EU alone, approximately 30 million people are affected by a rare disease, many of them children. Most of the 6,000 to 8,000 rare diseases known to date are caused by the altered function of a single gene (Boycott&Ardigó, 2018). This project under the supervision of Prof. Christoph Klein aims to develop innovative strategies for precision medicine in rare diseases by (i) re-wiring aberrant molecular networks for therapeutic purposes and (ii) identifying novel “druggable” targets using CRISPR-Cas9-mediated genome-wide screens.









