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CTRC Discovery of the Year Award.

Based on the following publication:
Crebbp haploinsufficiency in mice alters the bone marrow microenvironment, leading to loss of stem cells and excessive myelopoiesis.  Zimmer SN, Zhou Q, Zhou T, Cheng Z, Abboud-Werner SL, Horn D, Lecocke M, White R, Krivtsov AV, Armstrong SA, Kung AL, Livingston DM, Rebel VI.  Blood. 2011 Jul 7;118(1):69-79. Epub 2011 May 9.

Myelodysplastic syndrome (MDS) is a heterozygous group of hematopoietic stem cell (HSC) diseases, characterized by abnormal hematopoietic differentiation and a high propensity to progress to acute myeloid leukemia (AML).  In both children and adults, MDS can develop as a side effect of cancer therapy. With an increasing population of cancer survivors, it is expected that the incidence of MDS will increase. Yet, very little is known about this disease, including how to treat it effectively.  The Rebel’s laboratory developed a mouse model that displays all the characteristics of human MDS, including the high propensity to develop AML. 


In the 2011 Blood publication this mouse model was used to demonstrate that the bone marrow microenvironment facilitates MDS development and identified several potential molecular mediators.  This finding is of importance because currently the best treatment for MDS is a bone marrow transplant that replaces the patient’s hematopoietic system.


However, this treatment does not replace the microenvironment from the patient.  Thus, Dr Rebel’s work suggest that this may still cause significant hematopoietic problems, including relapse of the original disease.  This work lays the ground work to further investigate the role of the microenvironment in MDS with the intent to find alternative and better drugs to specifically manipulate the microenvironment in MDS patients.