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Non-planar perylene diimides dual-targeting mitochondrial membrane potential and mitochondrial DNA transcription for an-titumor Therapy

huo, j.; jin, x.; yin, m.; hu, n.; liu, t.; lu, w.; Tong, Q.; huang, j.; you, h.

2025-10-06 cancer biology
10.1101/2025.10.03.680392 bioRxiv
Show abstract

Mitochondrial DNA (mtDNA) transcription is an emerging target in cancer therapy. While enzyme-targeted inhibitors such as IMT1B block POLRMT, their organelle specificity remains unclear. Here, we report a distinct strategy using non-planar perylene diimide derivatives (PDIs). The fluorescent lead compound PDIC-BL combines mitochondrial membrane potential ({Delta}{Psi}m) dependent accumulation (validated in cells and isolated mitochondria), and with direct intercalation into mtDNA (Kd = 32 M), as demonstrated by single-molecule stretching assays. This dual-targeting mechanism leads to selective inhibition of mitochondrial transcription, ROS generation and triggers apoptosis. PDIC-BL exhibits superior antitumor efficacy in breast cancer cell lines (MCF-7, MDA-MB-231) and significantly supresses tumor growth in vivo, with good tolerability in cancer xenograft model in nude mice. This work not only elucidates the structure-activity relationship of non-planar PDIs, but also offers a generalizable strategy for developing organelle-selective DNA binding therapeutics.

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