DF/HCC Program Affiliation

Leukemia

Research Abstract

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.

Publications

• Lu J, Guo S, Ebert BL, Zhang H, Peng X, Bosco J, Pretz J, Schlanger R, Wang JY, Mak RH, Dombkowski DM, Preffer FI, Scadden DT, Golub TR.MicroRNA-mediated control of cell fate in megakaryocyte-erythrocyte progenitors.Dev Cell 2008 Jun;14(6):843-53.
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• Ebert BL, Galili N, Tamayo P, Bosco J, Mak R, Pretz J, Tanguturi S, Ladd-Acosta C, Stone R, Golub TR, Raza A.An erythroid differentiation signature predicts response to lenalidomide in myelodysplastic syndrome.PLoS Med 2008 Feb;5(2):e35.
  18271621
• Ebert BL, Pretz J, Bosco J, Chang CY, Tamayo P, Galili N, Raza A, Root DE, Attar E, Ellis SR, Golub TR.Identification of RPS14 as a 5q- syndrome gene by RNA interference screen.Nature 2008 Jan 17;451(7176):335-9.
  18202658