Mouse models of myelodysplastic syndromes.

Abstract

Myelodysplastic syndromes (MDS) are hematopoietic malignancies characterized by peripheral cytopenias in the face of normo- or hypercellular, dysplastic bone marrow that arise from mutations in the hematopoietic stem/progenitor cell (HSPC). The disease is characterized by multiple cytogenetic and molecular defects, which result in an extremely heterogeneous phenotype. Recently, significant efforts have been made to develop appropriate mouse models to study this complex disease. Because of the heterogeneity of MDS, no single model is able to capture the MDS phenotype in its entirety. In this review, we describe several MDS mouse models and discuss the advances made in our understanding of the different disease mechanisms within the malignant clone and the marrow microenvironment. In addition, we describe progress in xenotransplantation models of MDS and discuss questions that remain to be answered.