Benjamin Ebert, MD
We study the biology and treatment of cancer using hematopoiesis as a model system. The laboratory employs a range of genomic technologies as well as classical cellular and molecular biology approaches to investigate the biology of specific human diseases, particularly hematopoietic malignancies and disorders of red blood cell production.
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.
We are also actively involved in the identification and development of small molecules that could be useful for the treatment of cancer and hematologic disorders. We are studying compounds that induce fetal hemoglobin and could be useful for the sickle cell anemia. 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.