Pediatric myelodysplastic syndrome (MDS) is a pre-malignant bone marrow failure disorder of the hematopoietic system. Currently, the only curative option for MDS patients is a hematopoietic stem cell transplant, which carries significant risks, including severe morbidity and potentially life-threatening treatment-related complications. However, development of new therapies is limited by the insufficient understanding of the disease mechanisms.
In my research, I seek to uncover the underlying causes of pediatric MDS, identify novel therapeutic targets and gain deeper insight into why and how bone marrow failure occurs. By applying advanced (single-cell) sequencing techniques, such as CITE-seq and spatial transcriptomics, I aim to dissect the mechanisms driving MDS development and progression. These approaches will help to identify aberrant genes, pathways and interactions of the failing bone marrow – ultimately leading to deeper understanding of MDS and hopefully making a positive impact on the wellbeing and prognosis of children diagnosed with MDS.