A high-throughput screening platform to identify MYCN expression inhibitors for liver cancer therapy.

ABSTRACT: MYCN, an oncogene implicated in hepatocellular carcinoma (HCC), is predominantly expressed in cancer stem-like HCC cells. It drives tumorigenicity, metastasis, and therapeutic resistance. In this study, we hypothesized that the pharmacological inhibition of MYCN could represent a novel therapeutic strategy for HCC. To identify inhibitors of MYCN expression, we developed an unbiased, high-throughput screening platform. With this platform, we identified MI202 as a potent inhibitor of MYCN expression. MI202 significantly reduced MYCN promoter activity and mRNA levels in HCC cells, inhibiting cell proliferation, spheroid formation, and colony growth and promoting apoptosis. Notably, MI202 selectively inhibited the proliferation of HCC cells but not of normal hepatic cells, highli

SUBMITTER: Xu Y 

PROVIDER: S-EPMC11868045 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature