探寻治疗靶点和预后分子标志一直是MDS治疗研究的关键问题。circRNA是近年研究倍受关注的一类与肿瘤发生、发展和预后密切相关的ncRNA分子。我们前期研究提示circRUNX1在MDS患者中显著低表达，上调其表达可抑制细胞增殖，促进凋亡；生物信息学分析提示其可能通过miRNA及RBP途径影响基因表达、转录、表观遗传修饰进而影响MDS发生发展和预后。本项目采用Microarray 分析、慢病毒载体调控、RNA pull-down、RIP、荧光素酶实验、质谱分析、RNA测序及活体示踪等技术，体内外实验结合研究circRUNX1在MDS发生发展及预后中的作用及机制；比较健康供者、不同危险分层MDS患者circRUNX1表达水平，探讨其作为MDS疾病危险分层和发生发展标志的可行性。研究将为circRUNX1作为MDS治疗靶点、预后标志等潜在的临床应用和丰富血液肿瘤的精准诊疗提供理论依据。

Exploring therapeutic targets and prognosis markers are alway the main issue to improve efficiency of MDS treatment. CircRNA is a group of ncRNAs closely related to tumorigenesis, development and prognosis in recent years. Our previous results revealed that circRUNX1 was significanly low-expressed in MDS patients. The up-regulation of circRUNX1 could inhibit cell proliferation and promote apoptosis of MDS cells. Bioinformatics analysis suggested that circRUNX1 might affect gene expression, transcription, epigenetic modifications and the development and prognosis of MDS through RBP and miRNA-mRNA pathway. In this study, the potential role and mechanisms of circRUNX1 in MDS development and prognosis were studied with lentiviral vector regulation, RNA pull-down, RIP, Luciferase assay, Mass spectrometry,RNA sequencing and in vivo tracking techniques. To compare the circRUNX1 expression levels in healthy donor and MDS patients with different risk stratification to investigate the feasibility of circRUNX1 being a marker for risk stratification and disease development in MDS.This research will provide theoretical and experimental evidence for the potential clinical application of circRUNX1 as a target and prognostic marker of MDS treatment, as well as to enrich the precise diagnosis and treatment of hematological malignancies.