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Gene transfer to the liver

Part 1. Genetics

1.7. Gene Therapy

Basic Techniques and Approaches

  1. Katherine Parker Ponder

Published Online: 15 JUL 2005

DOI: 10.1002/047001153X.g107407

Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics

Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics

How to Cite

Ponder, K. P. 2005. Gene transfer to the liver. Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics. 1:1.7:102.

Author Information

  1. University of Washington School of Medicine, St. Louis, MO, USA

Publication History

  1. Published Online: 15 JUL 2005


The liver is an important organ for gene therapy. There are a large number of genes that are expressed in the liver whose deficiency results in a genetic disorder that might be treated by expressing the deficient protein in the liver. In addition, hepatitis B and C are very common, and might be treated with vectors that express small interfering RNAs that downregulate viral gene expression. Hepatocytes are readily accessible to viral or plasmid vectors that are injected systemically owing to the fenestrations in the blood vessels in the liver, which makes in vivo delivery feasible. Gene therapy has had considerable success in the liver in animal models. Both retroviral and AAV vectors have been delivered to animals with no obvious toxicity and have resulted in long-term expression in both small- and large-animal models. Although adenoviral vectors have achieved very high levels of expression in rodents, they have evoked substantial inflammation, and have not resulted in stable expression in most large-animal studies. Plasmid vectors have been effective, although efficient delivery to the hepatocyte remains a problem. One or more of these gene therapy approaches will likely translate into an effective therapy for patients in the near future.


  • retroviral vector;
  • AAV vector;
  • adenoviral vector;
  • plasmid vector;
  • SV40 vector;
  • hemophilia;
  • hepatitis;
  • mucopolysaccharidosis