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Glycyrrhetinic acid functionalized polyamidoamine dendrimers for targeted gene delivery in liver cancer
Meijun Wang1, Yujing Sang2, Xinyu Qin1
1School of Pharmacy, Engineering Center of Henan Province of Eucommia ulmoides Cultivation and Utilization, Henan University, Kaifeng, China. congyue@henu.edu.cn.
Abstract:
Gene therapy holds significant promise for the treatment of liver cancer. However, the development of safe and efficient gene delivery systems remains a critical challenge. Cationic polymers are widely utilized as gene carriers due to their high transfection efficiency, yet their application is often hindered by cytotoxicity and lack of target specificity. In this study, we present a one-step strategy to conjugate glycyrrhetinic acid (GA) to cationic polymers, aiming to reduce cytotoxicity and enhance liver-specific targeting. A series of GA-modified polyamidoamine (PPI) dendrimers were synthesized by varying the degree of GA substitution and were comprehensively characterized. The resulting GA-PPI dendrimers exhibited strong plasmid DNA (pDNA) binding and protection capabilities, forming stable GA-PPI/pDNA polyplexes. Notably, GA-PPI dendrimers with 5.10% GA substitution achieved a 5.1-fold enhancement in hepatocyte transfection efficiency (22.7% vs. 4.45% for unmodified PPI), while maintaining high cell viability (97.8% vs. 76.4%). In vivo, GA-PPI-3/pDNA polyplexes demonstrated superior antitumor efficacy in HepG2 xenograft models. These results underscore the potential of GA-functionalized PPI dendrimers as an effective and safe platform for targeted gene therapy in hepatocellular carcinoma.
Insights
Glycyrrhetinic acid (GA) modification of cationic polymers enhances liver cancer gene therapy. GA-conjugated polyamidoamine (PPI) dendrimers improve targeting and reduce toxicity for effective hepatocellular carcinoma treatment.
Area of Science:
- Biomaterials Science
- Nanotechnology
- Gene Therapy
Background:
- Gene therapy offers promise for liver cancer treatment, but efficient and safe gene delivery remains a challenge.
- Cationic polymers are effective gene carriers but suffer from cytotoxicity and poor targeting.
- Glycyrrhetinic acid (GA) modification is explored to improve polymer-based gene delivery systems.
Purpose of the Study:
- To develop a one-step strategy for conjugating GA to cationic polymers (PPI dendrimers).
- To reduce cytotoxicity and enhance liver-specific targeting of gene delivery vectors.
- To evaluate the efficacy of GA-modified PPI dendrimers for hepatocellular carcinoma gene therapy.
Main Methods:
- Synthesis and characterization of GA-modified polyamidoamine (PPI) dendrimers with varying GA substitution degrees.
- Assessment of pDNA binding and protection capabilities of GA-PPI dendrimers.
- In vitro evaluation of hepatocyte transfection efficiency and cytotoxicity.
- In vivo assessment of antitumor efficacy in HepG2 xenograft models.
Main Results:
- GA-PPI dendrimers demonstrated strong pDNA binding and formed stable polyplexes.
- A 5.10% GA substitution resulted in a 5.1-fold increase in hepatocyte transfection efficiency.
- Cell viability was significantly improved with GA-PPI dendrimers compared to unmodified PPI.
- GA-PPI-3/pDNA polyplexes showed superior antitumor efficacy in vivo.
Conclusions:
- GA-functionalized PPI dendrimers represent a promising platform for targeted gene therapy in liver cancer.
- This strategy effectively reduces cytotoxicity and enhances liver-specific gene delivery.
- The developed GA-PPI dendrimers offer a safe and efficient approach for hepatocellular carcinoma treatment.
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