Impaired lymphocyte development such as with severe combined immunodeficiency (SCID) or X-linked agammaglobulinemia (XLA) are major threats to afflicted individuals. For SCID, the curative treatment exists in the form of a haematopoietic stem cell donor, but such a donor is not always available. XLA patients are treated by repeated immunoglobulin administration. Bone marrow transplantation could be seen as a cure for both diseases, however, an allograft often results in Graft versus Host Disease and other comorbitities. An autologous bone marrow transplant with corrected genes, a.k.a. gene therapy, would be the ideal cure for these diseases. The main focus for gene therapy of my PhD project will be RAG2 and BTK, deficiencies in these genes lead to SCID and XLA respectively.
I completed my bachelor in Biology and Medical Laboratory Research at the Hogeschool Rotterdam and continued to complete both the premaster and master in Biomedical Sciences at the Leiden University. At the Leiden University I focused on immunology courses and graduated in the research specialisation in 2022. In my last masters research internship I joined the immunology department at the Erasmus Medical Centre where I worked on autoimmune disease in the group of associate prof. Marjan A. Versnel. After my master I joined the group of prof. Frank Staal as PhD student to work on gene therapy in immunodeficiencies.