Medulloblastoma (MB) is a clinically challenging, childhood brain tumor with a diverse genetic makeup and differential miRNA profile. Aiming to identify deregulated miRNAs in MB, the miRNA expression profile of human MB samples was compared to that of normal cerebellar tissues. As a result, 8 upregulated and 64 downregulated miRNAs were identified in MB samples. Although various algorithms have been developed to predict the interaction between miRNA-mRNA pairs, the complexity and fidelity of miRNA-mRNA remain a concern. Therefore, to identify the signatures of miRNA-mRNA interactions essential for MB pathogenesis, miRNA profiling, RNA sequencing, and ingenuity pathway analysis (IPA) were performed in the same primary human MB samples. Further, when miR-217 was inhibited, a significant upregulation of predicted target genes SIRT1, ROBO1, FOXO3, and SMAD7 in HDMB03 cells was observed, confirming the validity of our approach. Functional analysis revealed that the inhibition of miR-217 in HDMB03 cells suppresses colony formation, migration, invasion, promoted apoptosis, and arrested cell population in S phase, indicating that manipulation of miR-217 may have a therapeutic potential for MB patients. Therefore, our study provides an essential platform for future investigations of specific miRNAs responsible for MB pathogenesis.

髓母细胞瘤（MB）是一种临床上具有挑战性的儿童期脑肿瘤，具有多种遗传组成和不同的miRNA谱。为了鉴定MB中失调的miRNA，将人MB样品的miRNA表达谱与正常小脑组织的miRNA表达谱进行了比较。结果，在MB样品中鉴定出8种上调的miRNA和64种下调的miRNA。尽管已经开发出各种算法来预测miRNA-mRNA对之间的相互作用，但miRNA-mRNA的复杂性和保真度仍然令人担忧。因此，为了鉴定对于MB发病机理必不可少的miRNA-mRNA相互作用的特征，在相同的原始人MB样品中进行了miRNA分析，RNA测序和智能路径分析（IPA）。此外，当miR-217被抑制时，预测的目标基因SIRT1，ROBO1，FOXO3，在HDMB03细胞中观察到SMAD7和SMAD7，证实了我们方法的有效性。功能分析表明，在HDMB03细胞中抑制miR-217可以抑制菌落的形成，迁移，侵袭，促进细胞凋亡以及在S期抑制细胞群体，这表明操纵miR-217可能对MB患者具有治疗潜力。因此，我们的研究为负责MB发病机理的特定miRNA的未来研究提供了重要的平台。