The overarching aim of our research is to develop novel, cancer-specific, patient-orientated treatment options for children with acute leukemia.
Among the different types of leukemia, our research primarily focuses on acute myeloid leukemias, a subtype in which treatment outcomes have not substantially improved for pediatric patients for decades. We are particularly interested in children with Down syndrome, who are predisposed towards developing myeloid leukemia, and who present with increased sensitivity to treatment-related toxicities. Over the years, we have contributed to identifying coding and non-coding genes on chromosome 21 that are involved in disease pathogenesis. We also uncovered a complex transcriptional network that is deregulated during malignant transformation, at the center of which is GATA1s – a crucial factor for Down syndrome myeloid leukemia development. Utilizing advanced molecular biology approaches, we aim to delineate the interaction partners of GATA1s, the oncogenic network on chromosome 21, and co-occurring mutations during the transformation into Down syndrome myeloid leukemia.