神経芽腫におけるMYCN遺伝子を標的としたPIポリアミドの抗腫瘍効果に関する研究

神经母细胞瘤是最常见的儿童实体肿瘤。NB具有MYCN扩增的不良结局，MYCN表达下调导致NB的增殖下调和分化。我们设计了针对MYCN基因启动子区SP1和E2F结合位点的PI聚酰胺。在PI聚酰胺中培养NB细胞株，如CHP134细胞。与未经PI聚酰胺处理的NB细胞株相比，NB细胞株的增殖和MYCN表达均下调。我们利用CHP134细胞制备异种移植裸鼠。在NB异种移植小鼠中，我们给PI聚酰胺注入肿瘤。与未治疗组相比，肿瘤生长明显下降。该聚酰胺有望成为治疗MYCN基因扩增型神经母细胞瘤的新药物之一。

Neuroblastoma(NB) is the most common pediatric solid tumor. NB has MYCN amplified is poor outcome and down regulation of MYCN expression causes proliferation down-regulated and differentiation in NB. We designed PI polyamide targeting for SP1 and E2F binding site, which are promoter region of MYCN gene. NB cell lines, such as CHP134 cells were cultured in the PI polyamide. NB cell lines were down-regulation of proliferation and of MYCN expression, compared with NB cell lines untreated with PI polyamide. We made xenograft nude mice by using CHP134 cells. In NB xeno-graft mice, was we administrated PI polyamide into the tumor. Then the tumors growth was down-regulated, compared with non-treated group. This PI polyamide could be one of the new therapeutic agents for MYCN gene amplified neuroblastoma.