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Updated: May 30, 2026
09:54
Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
Perinatal gene transfer to the liver.
Tristan R McKay1, Ahad A Rahim, Suzanne M K Buckley
1William Harvey Research Institute, Queen Mary University of London, London, UK.
Current Pharmaceutical Design
|July 22, 2011
Summary
Early gene therapy shows promise for treating genetic liver diseases in newborns and fetuses. This review explores gene therapy tools and their application for various inherited liver conditions.
Area of Science:
- Hepatology
- Genetics
- Gene Therapy
Background:
- The liver performs numerous vital functions, making it susceptible to genetic defects.
- Gene mutations can cause a spectrum of liver diseases, from mild to fatal in early infancy.
- While some liver conditions are manageable, others remain untreatable with current medicines.
Purpose of the Study:
- To review the potential of early intervention gene therapy for neonatal and fetal liver diseases.
- To evaluate gene therapy tools and their efficacy in preclinical studies.
- To assess the application of gene therapy for specific inherited liver disorders.
Main Methods:
- Review of lentivirus, adenovirus, and adeno-associated virus (AAV)-based vectors for gene delivery.
- Analysis of preclinical studies on fetal and neonatal gene transfer to the liver.
- Examination of gene therapy applications for hemophilia, urea cycle disorders, organic acidemias, lysosomal storage diseases, glycogen storage diseases, and bile metabolism disorders.
Main Results:
- Gene therapy vectors like AAV show potential for correcting genetic defects in the liver.
- Preclinical studies demonstrate feasibility of fetal and neonatal gene transfer.
- Successful application in models of hemophilia, ornithine transcarbamylase deficiency, and other metabolic disorders.
Conclusions:
- Early gene therapy offers a potential treatment strategy for severe genetic liver diseases.
- Fetal and neonatal gene transfer present unique advantages and challenges.
- Further research is needed to optimize safety and efficacy for clinical translation.
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