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Gene Delivery to the Liver

Karen Kozarsky · Current Protocols in Human Genetics · 1999

AbstractViral 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.

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