We are among the few labs worldwide that have successfully transitioned from fundamental research to translational and clinical applications, actively preparing for clinical trials. Notably, we initiated the world's first clinical trial for RAG1 SCID—a severe immunodeficiency where gene therapy has historically faced significant efficacy challenges. Patient involvement is central to our work, and we collaborate closely with patient advocacy groups, including IPOPI and the Dutch SAS, through our scientific grants.
Beyond our research, we are committed to public engagement and education. Our dissemination efforts include lectures for general audiences, media appearances (radio, TV, newspapers, and websites such as https://www.lumc.nl/projecten/cure4life/; cure4life), patient information leaflets, and postgraduate courses for MDs and nurses.
