Related Experiment Video
Updated: May 30, 2026

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
Gene therapy of liver diseases: a 2011 perspective
Gloria Gonzalez-Aseguinolaza1, Jesús Prieto
1Division of Hepatology and Gene Therapy, Centro de Investigación Medica Aplicada and Clinica Universitaria, University of Navarra, Pamplona, Spain.
Abstract:
Liver diseases including inherited metabolic disorders, chronic viral hepatitis, liver cirrhosis and primary and metastatic liver cancer constitute a formidable health problem because of their high prevalence and the important limitations of current therapies. Gene therapy, a procedure based on the transfer of therapeutic genes to tissues, has been used since the 1990s as a new approach to treating a number of incurable conditions. After a period of lights and shades recent success in treating several devastating diseases like inherited immune deficiency disorders, beta-thalassemia, or inherited blindness appear to herald a new era where gene therapy can be listed among standard therapy options for a wide variety of human conditions. In this review, we provide information illustrating the potentiality of gene therapy in the management of liver diseases lacking other effective therapies.
Insights
Gene therapy offers a promising new approach for treating liver diseases, including cancer and inherited disorders, where current treatments are limited. Recent advancements suggest gene therapy may soon become a standard option for various liver conditions.
Area of Science:
- Hepatology
- Molecular Biology
- Medical Genetics
Background:
- Liver diseases pose significant global health challenges due to high prevalence and limited therapeutic options.
- Current treatments for conditions like cirrhosis, viral hepatitis, and liver cancer have notable limitations.
- Gene therapy has emerged as a novel therapeutic strategy since the 1990s.
Purpose of the Study:
- To review the potential of gene therapy for managing liver diseases.
- To highlight recent successes and future prospects of gene therapy in hepatology.
- To explore gene therapy as an alternative for incurable liver conditions.
Main Methods:
- Literature review of gene therapy applications in liver disease.
- Analysis of recent clinical successes and ongoing research in the field.
- Synthesis of information on gene transfer techniques for therapeutic purposes.
Main Results:
- Gene therapy has shown success in treating other severe genetic disorders, indicating its potential for liver diseases.
- Recent advancements suggest gene therapy is moving towards becoming a standard therapeutic option.
- The review illustrates the significant potential of gene therapy for liver conditions lacking effective treatments.
Conclusions:
- Gene therapy represents a promising frontier for treating a wide range of liver diseases.
- Further research and clinical application of gene therapy could revolutionize liver disease management.
- The potential for gene therapy to address unmet needs in liver disease is substantial.
Related Concept Videos
Effect of Hepatic Disease on Pharmacokinetics: Pathophysiologic Assessment and Liver Function Test
Gene Therapy
Gene Therapy
Pharmacogenomics: Identification of New Drug Targets
Diseases of the Liver and Gallbladder
Cirrhosis is characterized by the scarring of hepatic lobules in the liver, which are replaced by fibrous tissue, affecting the liver's normal functioning. NAFLD, on the other hand, is caused by an excessive build-up of fat in the liver, not related to...
Effect of Hepatic Disease on Pharmacokinetics: Drug Dosing and Hepatic Blood Flow
