UNIT 13.10 Gene Delivery to the Liver

  1. Karen Kozarsky

Published Online: 1 MAY 2001

DOI: 10.1002/0471142905.hg1310s22

Current Protocols in Human Genetics

Current Protocols in Human Genetics

How to Cite

Kozarsky, K. 2001. Gene Delivery to the Liver. Current Protocols in Human Genetics. 22:13.10:13.10.1–13.10.7.

Author Information

  1. SmithKline Beecham Pharmaceuticals, Inc., King of Prussia, Pennsylvania

Publication History

  1. Published Online: 1 MAY 2001
  2. Published Print: AUG 1999


Viral gene transfer to the liver has proven extremely effective in animal models and is currently being evaluated in clinical trials for a variety of metabolic disorders. In rodents, a single tail vein injection of an adenoviral vector can transduce most hepatocytes in vivo. This provides a convenient model for assessing vector design as well as for evaluating the effects of specific transgenes in genetic mouse models of human disease. Protocols for optimized in vivo hepatic gene transfer are described.