The research group led by Frank Staal has developed a gene therapy approach for patients with RAG1-SCID, a condition caused by mutations in the RAG1 gene that completely block the development of B and T cells. Using a lentiviral vector designed to deliver a healthy copy of the RAG1 gene into HSCs, the team has successfully treated six RAG1-SCID patients to date.
While this viral delivery method is very effective, controlling where and how strongly the gene is switched on remains a challenge. The current research focuses on making gene therapy safer by adding genetic “control switches” that limit gene activity to specific immune cells. As a first step, we aim to restrict gene expression to B cells, paving the way for safer treatments for other immune diseases such as X-linked agammaglobulinemia (XLA).