We study the biology and treatment of cancer using hematopoiesis as a model system. We combine genomic analyses of patient samples with functional screens to identify critical genes for hematologic malignancies, hematopoietic differentiation, and hematopoietic stem cell self renewal. In addition, we are working on the identification and characterization of compounds that alter hematopoietic differentiation that could be useful for the treatment of cancer and non-malignant hematopoietic disorders.
A major focus of the laboratory is the myelodsyplastic syndrome (MDS), a pre-malignant disorder of hematopoietic stem cells that progresses to acute leukemia. In recent work, we identified a gene that plays a central role in the pathophysiology of the 5q- syndrome, a subtype of MDS. Our findings revealed a molecular link between the 5q- syndrome and congenital bone marrow failure syndromes such as Diamond Blackfan Anemia.