Professor Frank Staal works on a gene therapy for patients, often young children, suffering from severe combined immunodeficiency (SCID). Their immune system does not work properly and in some cases not at all. This leaves them vulnerable to all types of pathogens. Starting with a specific disease type called RAG1-SCID, Staal contributed to the first in man clinical trial of a gene therapy for this disease. Together with his team, he now works on making this stem cell-based therapy more widely available, for example by addressing scalability. They also aim for a standardized monitoring of clinical trials for these products, and on extending their gene technique for other types of immune deficiencies.

Professor Staal is a reNEW principal investigator at the Department of Immunohematology at Leiden University Medical Center. In 2007, he was appointed professor at Leiden University Medical School and moved his lab from Rotterdam to Leiden. He has many international collaborators and teaches at several universities in the Netherlands. Professor Staal is coordinator of two large European consortiums focusing on gene therapy for SCID. In addition, he is an editor at Future Science OA and Leukemia, as well as reviewer for all major biomedical journals. His work has been published in Nature, Nature Reviews Immunology, Immunity, Cell Stem Cell, and other prestigious journals. He is regularly invited to hold lectures and presentations to scientists, patient advocate groups and laymen audiences.