Department of Cancer and Cell Biology, College of Medicine, University of Cincinnati, Cincinnati, OH, USA.
Leukemia. 2012 Jan;26(1):13-22. doi: 10.1038/leu.2011.221. Epub 2011 Aug 19.
Myelodysplastic syndromes (MDSs) consist of a family of hematopoietic stem cell (HSC) disorders characterized by ineffective differentiation of hematopoietic progenitors, bone marrow dysplasia, genetic instability and a propensity to develop acute myeloid leukemia. The development of MDS is poorly understood and therefore, effective treatment options are limited. Recent progress has been made in identifying altered signaling pathways and understanding the HSC defects, which are thought to contribute to the pathogenesis of MDS. Several of these findings have implicated aberrant expression and function of microRNAs (miRNAs). Unique miRNA expression patterns have been identified in MDS patients and modeled in mice to recapitulate features of MDS. Here, we review miRNA expression profiles identified in MDS patients, and describe the association of miRNA expression with MDS subtypes and disease outcome, clinical implications of miRNAs in MDS and deregulation of miRNAs in mouse model systems of MDS.
骨髓增生异常综合征（MDSs）是一组造血干细胞（HSC）疾病，其特征为造血祖细胞分化无效、骨髓增生不良、遗传不稳定性以及发展为急性髓系白血病的倾向。MDS 的发病机制尚不清楚，因此有效的治疗选择有限。最近在确定改变的信号通路和理解 HSC 缺陷方面取得了进展，这些缺陷被认为有助于 MDS 的发病机制。其中一些发现涉及 microRNAs（miRNAs）的异常表达和功能。在 MDS 患者中已经鉴定出独特的 miRNA 表达模式，并在小鼠中进行建模以重现 MDS 的特征。在这里，我们综述了 MDS 患者中鉴定出的 miRNA 表达谱，并描述了 miRNA 表达与 MDS 亚型和疾病结局的关联、miRNAs 在 MDS 中的临床意义以及 MDS 小鼠模型系统中 miRNAs 的失调。
