Analysis of JAK-inhibitor treatment for STAT1 gain-of-function and development of a CRISPR-based gene therapy approach KU Leuven
Primary immunodeficiencies (PIDs) are rare inherited disorders affecting the immune system presenting with increased susceptibility to pathogens and often associated with severe non-infectious comorbidities. The poor understanding of PIDs makes them difficult to diagnose and thus, challenging to treat or cure. The monogenic nature of several PIDs converts them in the perfect target for precision medicine. In this project, I focus in autosomal ...