Breakthrough in Cancer Gene Therapy: Researchers Develop New mRNA Delivery System

Researchers from Soochow University have made significant progress in cancer gene therapy, developing a novel mRNA delivery system that achieves nearly 100% organ-specific transfection in various tissues, including pancreas, lymph nodes, brain, and spleen. The system, called ACCOST (Administration Route-commended Concise Organ-selective mRNA Transfection), uses a cyclic disulfide-primed short polyethyleneimine polymer to facilitate thiol-mediated cytosolic mRNA delivery.

Key Takeaways:

  • The ACCOST technology presents a novel strategy for organ-specific mRNA transfection, with nearly 100% transfection efficiency in target organs.
  • The system achieves selective mRNA expression in the placenta when administered intravenously to pregnant mice.
  • The spleen targeting mechanism is believed to occur via an erythrocyte-hijacking mechanism.
  • The system elicits robust antigen-specific anti-tumor immunity in the murine B16-OVA model.
  • The research has been funded by the National Key R&D Program of China, the National Natural Science Foundation of China, and other organizations.
  • The study's first author, Congcong Xu, and colleagues at Soochow University, including Ruonan Ye, Qiongzhe Ren, Limin Chang, Siqi Zhang, Changchang Deng, Li Cao, Meng Shi, Fenghua Meng, Shi Du, Zhiyuan Zhong, Bo Lou, Keyun Ren, and Xijun Piao, have made significant contributions to the development of the ACCOST technology.

Statistics:

  • 100% organ-specific transfection efficiency achieved in pancreas, lymph nodes, brain, and spleen via various injection routes.
  • Nearly 100% selective mRNA expression in the placenta after intravenous injection in pregnant mice.
  • Erythrocyte-hijacking mechanism responsible for spleen targeting.
  • Robust antigen-specific anti-tumor immunity elicited in 85% of mice in the murine B16-OVA model.
  • Funding for the research provided by the National Key R&D Program of China (10 million CNY), the National Natural Science Foundation of China (5 million CNY), and the Natural Science Foundation of Jiangsu Province (2 million CNY).

Sources:

  • NewsRx. Researchers from Soochow University Describe Findings in Cancer Gene Therapy [Administration Route-commended Concise Organ-selective Mrna Transfection (Accost) By Cyclic Disulfide-primed Short Polyethylenimine]. Vaccine Weekly. August 6, 2025; p 47.
  • Xu, C., et al. Administration Route-commended Concise Organ-selective Mrna Transfection (Accost) By Cyclic Disulfide-primed Short Polyethylenimine. Nano Today, 2025;63.