New Approach for Liver Cancer Treatment and Possible Prevention: Gene Therapy Targets Biomarker for Hepatocellular Carcinoma (HCC)
Researchers at the UC Davis Comprehensive Cancer Center have unveiled a promising gene therapy approach that significantly reduces the incidence of hepatocellular carcinoma (HCC) in animal models, specifically mice.

This innovative research targets a specific protein known as galectin 1 (Gal1), which has long been established as a critical biomarker for HCC. By confirming the therapeutic potential of Gal1 as a target, the study paves the way for novel treatment strategies against liver cancer.
In this study, scientists employed an adeno-associated viral 9 vector (AAV9) to deliver a short interfering RNA (siRNA) called Igals1. This siRNA is designed to silence Gal1, thereby inhibiting its expression. AAV9 is particularly advantageous for liver therapies due to its inherent ability to effectively deliver transgenes specifically to liver cells, making it an ideal vehicle for targeted gene therapy.
The inhibition of Gal1 not only resulted in a significant reduction in HCC tumor size but also boosted anti-cancer immune responses. The therapy increased the infiltration of killer T-cells at the tumor site, enhancing the body’s immune response against cancerous cells. Importantly, this approach demonstrated efficacy not only in treating existing HCC tumors but also in preventing their development through the inhibition of Gal1.
Furthermore, the study highlights the dual role of galectin 1 in liver cancer progression. Gal1 not only acts as a biomarker but also plays an active role in the tumor microenvironment by promoting cancer cell survival and metastasis. The researchers believe that by targeting Gal1, they could disrupt these pathways, ultimately leading to more effective cancer treatment.
As this research advances, it holds the potential to revolutionize HCC management, offering hope for better outcomes in patients at risk of developing liver cancer.
Video describing how gene therapy can be used to treat cancers.
(Media source: https://www.youtube.com/watch?v=DNf2GsjfRQk)
Created: February 2nd, 2024
Citation:
Tahereh S., Ying H., Farzam V., Xin C., Jinping L., Dongguang W., Yu-Jui Y. W. Targeting stroma and tumor, silencing galectin 1 treats orthotopic mouse hepatocellular carcinoma. Acta Pharmaceutica Sinica B Volume 14 Issue 1, 292-303 (2024). https://doi.org/10.1016/j.apsb.2023.10.010
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