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首页> 美国政府科技报告 >Functional Role of MicroRNAs in Hematopoietic Stem Cells in the Myelodysplastic Syndromes.
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Functional Role of MicroRNAs in Hematopoietic Stem Cells in the Myelodysplastic Syndromes.

机译：微小RNa在骨髓增生异常综合征中的造血干细胞中的功能作用。

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摘要

Myelodysplastic syndromes (MDS) are acquired bone marrow failure disorders characterized by poor clinical outcomes due to progressively worsening blood cell counts (especially red blood cells) and increased risk of developing leukemia. MDS originates in hematopoietic stem cells (HSC), but there is little information available regarding the molecular mechanisms regulating MDS HSC function. We have characterized the expression of more than 750 microRNAs in MDS HSC purified from MDS patient samples and compared their expression to normal, age-matched controls. These studies identify at least 31 microRNAs that are differentially expressed in MDS HSC compared to normal controls (FDR > 0.1, P < 0.05). We have initiated studies to characterize the potential role of these microRNAs in MDS pathogenesis by using a combination of in vitro assays and a newly developed xenograft model of MDS. Our findings are consistent with a role of miRNAs in MDS HSC function and future studies will aim to validate the role of each of these candidate miRNAs in MDS development and progression.

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