Research group Molecular hemato-oncology

Aims

• To identify therapeutic targets and develop small molecules to cure blood cancer and bleeding disorders.

Changes in gene expression play a dominant role in the development of many blood cell disorders. These changes are caused by inherited and acquired mutations in transcription factors and epigenetic modifiers. Blood cell disorders also arise through changes in expression of nonmutated genes that affect chromatin biology.

My group determines how transcription factors and epigenetic modifiers affect gene expression at the molecular level and which gene programs are relevant for disease pathogenesis. Insight into these processes provide new targets for therapy. Based on our findings, small molecules are being synthesized that specifically correct malfunctioning proteins to normalize affected blood cell development.

Achievements

1. We uncovered genetic and epigenetic mechanisms that disrupt blood-cell development and contribute to inherited bleeding disorders and hematological malignancies.
2. We developed and implemented molecular approaches that improve disease diagnosis, classification, risk assessment and treatment monitoring.
3. We established advanced disease models and computational approaches to identify therapeutic vulnerabilities and develop strategies for precision treatment.

Publications

See the publication list of the research group leader on Web of Science.

• A selection of our publications

  • Monteferrario D, et al. A Dominant-Negative GFI1B Mutation in the Gray Platelet Syndrome. N Engl J Med. 2014;370:245-253.
  • Chen L, et al. Transcriptional diversity during lineage commitment of human blood progenitors. Science. 2014;345:1251033.
  • Langemeijer SMC, et al. Acquired mutations in TET2 are common in myelodysplastic syndromes. Nat Genet. 2009;41:838-842.
  • Cloos J, et al. 2025 update on measurable residual disease in acute myeloid leukemia. Blood. 2026;147:1147-1167.
  • Van Zeventer IA, et al. Evolutionary landscape of clonal hematopoiesis in 3,359 individuals from the general population. Cancer Cell. 2023.
  • Van Bergen MGJM, et al. Specific proteome changes in platelets from individuals with GATA1-, GFI1B- and RUNX1-linked bleeding disorders. Blood. 2021;138:86-90.
  • Arza-Apalategi S, et al. HMX3 is a critical vulnerability in MECOM-negative KMT2A::MLLT3 acute myelomonocytic leukemia. Leukemia. 2025;39:371-380.
  • Da Silva-Coelho P, et al. Clonal evolution in myelodysplastic syndromes. Nat Commun. 2017;8:15099.
  • Van Oorschot R, et al. Molecular mechanisms of bleeding disorder-associated GFI1B Q287* mutation and its affected pathways in megakaryocytes and platelets. Haematologica. 2019.
  • Nikoloski G, et al. Somatic mutations of the histone methyltransferase gene EZH2 in myelodysplastic syndromes. Nature Genetics. 2010;42:665-667.