Gene Therapy with Enterovirus 3 C Protease: A Promising Strategy for Various Solid Tumors

Abstract Current cancer gene therapies rely primarily on antitumor immunity, but the exploration of alternative mRNA cargoes for direct antitumor effects is crucial to expand cancer gene therapies. Here we show that lipid nanoparticles (LNPs) carrying mRNA encoding a viral 3 C protease can efficient...

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Main Authors: Xiaotong Yang, Wei Li, Shaokang Yang, Zhuang Wang, Jiye Yin, Wenhao Zhang, Huimin Tao, Siqi Li, Xiaojia Guo, Qingsong Dai, Weiyan Zhu, Yuexiang Li, Xintong Yan, Chongda Luo, Jiazheng Li, Sichen Ren, Ping Wang, Yunfeng Shao, Yan Luo, Zhenyang Li, Jingjing Yang, Zhijie Chang, Ruiyuan Cao, Song Li, Wu Zhong
Format: Article
Language:English
Published: Nature Portfolio 2025-05-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-59440-8
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Summary:Abstract Current cancer gene therapies rely primarily on antitumor immunity, but the exploration of alternative mRNA cargoes for direct antitumor effects is crucial to expand cancer gene therapies. Here we show that lipid nanoparticles (LNPs) carrying mRNA encoding a viral 3 C protease can efficiently suppress tumors by selectively inducing tumor cell apoptosis. In various solid tumor models, intracranial injection of LNPs carrying mRNA encoding the 3 C protease (3C-LNPs) significantly inhibits tumor growth and prolongs survival in glioblastoma models. Similarly, subcutaneous injection reduces tumor volume and inhibits angiogenesis in a breast cancer model, while intravenous injection inhibits tumor growth and angiogenesis and prolongs survival in hepatocellular carcinoma models. Mass spectrometry and cleavage site prediction assays identify heterogeneous nuclear ribonucleoprotein A1 (hnRNP A1) as the main target degraded by the 3 C protease. This study suggests that viral protease mRNA could be a promising broad-spectrum antitumor therapeutic.
ISSN:2041-1723