Neuroblastoma (NB) is one of the most common malignant tumors in children. Chemotherapy resistance is one of the significant challenges in the treatment of high-risk NB patients, and it is necessary to search for new valid targets for NB treatment. This study aims to explore the possible role of PIF1 in NB by using bioinformatic analysis and downregulation of PIF1 with specific siRNA. Kyoto genome encyclopedia and R language based gene ontology was used to analyze the differentially expressed genes (DEGs) (including PIF1) when MYCN expression was silenced in NB cells. Analysis based on the R2 database showed a lower expression of PIF1 correlated with good prognosis in NB patients. Downregulation of MYCN expression by transfecting MYCN siRNA (#1, #2) into NB cells decreased the PIF1 expression at both mRNA and protein levels, while upregulation of MYCN expression by transfecting MYCN overexpressed plasmid increased the PIF1 expression. We further found that downregulation of PIF1 expression by transfecting PIF1 siRNA (#1, #2) into NB cells, increased the number of apoptotic cells, inhibited the cell survival, decreased the ability of cell migration and induced a cell cycle arrest at G1 phase. These data indicated that PIF1, as a potential new target of MYCN, maybe a novel target for NB treatment.

神经母细胞瘤（NB）是儿童中最常见的恶性肿瘤之一。化疗耐药是治疗高危NB患者的重大挑战之一，因此有必要寻找新的有效NB治疗靶点。这项研究旨在通过生物信息学分析和用特定siRNA下调PIF1来探讨PIF1在NB中的可能作用。京都基因组百科全书和基于R语言的基因本体用于分析NB细胞中MYCN表达沉默时的差异表达基因（DEG）（包括PIF1）。基于R2数据库的分析显示NB患者的PIF1表达较低，与预后良好相关。通过将MYCN siRNA（＃1，＃2）转染到NB细胞中，MYCN表达下调降低了mRNA和蛋白水平的PIF1表达，而通过转染MYCN过表达的质粒上调MYCN表达可增加PIF1表达。我们进一步发现通过将PIF1 siRNA（＃1，＃2）转染到NB细胞中来下调PIF1表达，增加凋亡细胞的数量，抑制细胞存活，降低细胞迁移的能力并诱导细胞周期停滞在G1期。这些数据表明，PIF1作为MYCN的潜在新靶标，可能是NB治疗的新靶标。