Low-liver-accumulation lipid nanoparticles enhance the efficacy and safety of HPV therapeutic tumor vaccines

Abstract Background Lipid nanoparticles (LNPs) hold significant potential in vaccine and cancer therapy, but conventional LNPs often cause hepatotoxicity and reduced efficacy due to liver accumulation. Methods LNP molecules with varying tail lengths were synthesized in vitro. Transfection efficiency...

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Main Authors: Xiaodie Wang, Zhidan Tian, Meng Wang, Xiaoru Cai, Shuyan Yang, Jianyao Zeng, Yuan Fang, Xuesen Bai, Pei Wang, Yan Sun, Chuanxin Guo, Tao Liu, Qijun Qian
Format: Article
Language:English
Published: BMC 2025-08-01
Series:Journal of Translational Medicine
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Online Access:https://doi.org/10.1186/s12967-025-06924-2
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author Xiaodie Wang
Zhidan Tian
Meng Wang
Xiaoru Cai
Shuyan Yang
Jianyao Zeng
Yuan Fang
Xuesen Bai
Pei Wang
Yan Sun
Chuanxin Guo
Tao Liu
Qijun Qian
author_facet Xiaodie Wang
Zhidan Tian
Meng Wang
Xiaoru Cai
Shuyan Yang
Jianyao Zeng
Yuan Fang
Xuesen Bai
Pei Wang
Yan Sun
Chuanxin Guo
Tao Liu
Qijun Qian
author_sort Xiaodie Wang
collection DOAJ
description Abstract Background Lipid nanoparticles (LNPs) hold significant potential in vaccine and cancer therapy, but conventional LNPs often cause hepatotoxicity and reduced efficacy due to liver accumulation. Methods LNP molecules with varying tail lengths were synthesized in vitro. Transfection efficiency was assessed in vitro via flow cytometry. The BALB/c model was used to evaluate in vivo delivery efficiency of the LNPs. The C57BL/6 mouse model was used to evaluate in vivo anti-tumor pharmacodynamics, along with tumor-infiltrating immune cells and serum inflammatory cytokines. The Sprague Dawley (SD) rat model was used for toxicity testing of the candidate LNP. Results Lipid 7 demonstrated threefold higher mRNA expression efficiency at the injection site while minimizing liver retention. In an HPV tumor model, Lipid 7 achieved tumor suppression comparable to SM-102-based LNP but outperformed in remodeling the tumor microenvironment (dendritic cells: 12.1% vs. 5.1%; natural killer cells: 1.1% vs. 0.5%) and elevating serum immune cytokines (TNF-α, IL-1β, etc., 1.2–1.8-fold higher). Critically, Lipid 7 reduced off-target mRNA accumulation in the heart, liver, spleen, lungs, and kidneys, mitigating hepatotoxicity risks associated with traditional LNPs. Conclusions By employing a novel, non-patented ionizable lipid design, this work balances delivery efficiency and biosafety, Lipid 7 offers a high-efficacy, low-toxicity therapeutic strategy for HPV-related cancers. Graphical Abstract
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publishDate 2025-08-01
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spelling doaj-art-886e63a4aa5f43d7a4f20cfad59da7d82025-08-20T03:46:24ZengBMCJournal of Translational Medicine1479-58762025-08-0123111510.1186/s12967-025-06924-2Low-liver-accumulation lipid nanoparticles enhance the efficacy and safety of HPV therapeutic tumor vaccinesXiaodie Wang0Zhidan Tian1Meng Wang2Xiaoru Cai3Shuyan Yang4Jianyao Zeng5Yuan Fang6Xuesen Bai7Pei Wang8Yan Sun9Chuanxin Guo10Tao Liu11Qijun Qian12School of Medicine, Shanghai UniversityShanghai Cell Therapy Group Co., LtdShanghai Cell Therapy Group Co., LtdShanghai Cell Therapy Group Co., LtdShanghai Cell Therapy Group Co., LtdShanghai Cell Therapy Group Co., LtdSchool of Medicine, Shanghai UniversityShanghai Cell Therapy Group Co., LtdSchool of Medicine, Shanghai UniversitySchool of Medicine, Shanghai UniversityShanghai Cell Therapy Group Co., LtdShanghai Cell Therapy Group Co., LtdSchool of Medicine, Shanghai UniversityAbstract Background Lipid nanoparticles (LNPs) hold significant potential in vaccine and cancer therapy, but conventional LNPs often cause hepatotoxicity and reduced efficacy due to liver accumulation. Methods LNP molecules with varying tail lengths were synthesized in vitro. Transfection efficiency was assessed in vitro via flow cytometry. The BALB/c model was used to evaluate in vivo delivery efficiency of the LNPs. The C57BL/6 mouse model was used to evaluate in vivo anti-tumor pharmacodynamics, along with tumor-infiltrating immune cells and serum inflammatory cytokines. The Sprague Dawley (SD) rat model was used for toxicity testing of the candidate LNP. Results Lipid 7 demonstrated threefold higher mRNA expression efficiency at the injection site while minimizing liver retention. In an HPV tumor model, Lipid 7 achieved tumor suppression comparable to SM-102-based LNP but outperformed in remodeling the tumor microenvironment (dendritic cells: 12.1% vs. 5.1%; natural killer cells: 1.1% vs. 0.5%) and elevating serum immune cytokines (TNF-α, IL-1β, etc., 1.2–1.8-fold higher). Critically, Lipid 7 reduced off-target mRNA accumulation in the heart, liver, spleen, lungs, and kidneys, mitigating hepatotoxicity risks associated with traditional LNPs. Conclusions By employing a novel, non-patented ionizable lipid design, this work balances delivery efficiency and biosafety, Lipid 7 offers a high-efficacy, low-toxicity therapeutic strategy for HPV-related cancers. Graphical Abstracthttps://doi.org/10.1186/s12967-025-06924-2mRNA deliveryLipid nanoparticles (LNPs)Cancer immunotherapyHepatotoxicity mitigation
spellingShingle Xiaodie Wang
Zhidan Tian
Meng Wang
Xiaoru Cai
Shuyan Yang
Jianyao Zeng
Yuan Fang
Xuesen Bai
Pei Wang
Yan Sun
Chuanxin Guo
Tao Liu
Qijun Qian
Low-liver-accumulation lipid nanoparticles enhance the efficacy and safety of HPV therapeutic tumor vaccines
Journal of Translational Medicine
mRNA delivery
Lipid nanoparticles (LNPs)
Cancer immunotherapy
Hepatotoxicity mitigation
title Low-liver-accumulation lipid nanoparticles enhance the efficacy and safety of HPV therapeutic tumor vaccines
title_full Low-liver-accumulation lipid nanoparticles enhance the efficacy and safety of HPV therapeutic tumor vaccines
title_fullStr Low-liver-accumulation lipid nanoparticles enhance the efficacy and safety of HPV therapeutic tumor vaccines
title_full_unstemmed Low-liver-accumulation lipid nanoparticles enhance the efficacy and safety of HPV therapeutic tumor vaccines
title_short Low-liver-accumulation lipid nanoparticles enhance the efficacy and safety of HPV therapeutic tumor vaccines
title_sort low liver accumulation lipid nanoparticles enhance the efficacy and safety of hpv therapeutic tumor vaccines
topic mRNA delivery
Lipid nanoparticles (LNPs)
Cancer immunotherapy
Hepatotoxicity mitigation
url https://doi.org/10.1186/s12967-025-06924-2
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