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Updated: Aug 3, 2026

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
Gene therapy of hepatocellular carcinoma
J Ruiz1, G Mazzolini, B Sangro
1Division of Hepatology and Gene Therapy, Department of Medicine, School of Medicine, University of Navarra, Pamplona, Spain. jruiz@unav.es
Abstract:
The extraordinary versatility of gene therapy opens new possibilities for the treatment of incurable diseases, including hepatocellular carcinoma. Gene therapy strategies against tumors include prodrug activation therapy by the transfer of suicide genes, immunogene therapy, tumoral cell phenotype correction by the inhibition of oncogenes or the transfer of tumor suppressor genes, antiangiogenesis and transfer of oncolytic viruses. The experience accumulated during the last decade of clinical gene therapy indicates that genes can be expressed inside the tumor tissue, but the overall results of the studies conducted so far are still disappointing, mainly due to the poor performance of the currently available gene therapy vectors. This review covers the general aspects of gene therapy vectors, preclinical data available in animal models of hepatocellular carcinoma, and finally a brief summary of the gene therapy clinical trials aimed at the treatment of liver cancer.
Insights
Gene therapy offers novel treatments for liver cancer, but current vector technology limits effectiveness. Research explores various strategies and clinical trials to improve outcomes for hepatocellular carcinoma.
Area of Science:
- Oncology
- Molecular Biology
- Biotechnology
Background:
- Hepatocellular carcinoma (liver cancer) remains a significant global health challenge with limited treatment options.
- Gene therapy presents a versatile approach for treating incurable diseases, including liver cancer.
Purpose of the Study:
- To review gene therapy strategies for hepatocellular carcinoma.
- To evaluate preclinical data and summarize clinical trials for liver cancer gene therapy.
Main Methods:
- Review of existing literature on gene therapy vectors.
- Analysis of preclinical studies in animal models of hepatocellular carcinoma.
- Summary of clinical trial outcomes for liver cancer gene therapy.
Main Results:
- Gene therapy employs diverse strategies like suicide gene therapy, immunogene therapy, and oncolytic viruses.
- While gene expression in tumors is achievable, current gene therapy vectors show poor performance.
- Clinical trial results for liver cancer gene therapy have been largely disappointing.
Conclusions:
- Despite challenges with current vectors, gene therapy holds promise for hepatocellular carcinoma treatment.
- Further advancements in gene therapy vector technology are crucial for improving clinical efficacy.
- Ongoing research and clinical trials are essential to realize the full potential of gene therapy for liver cancer.
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