Current techniques for the alteration of gene expression in the liver have a number of
limitations, including the lack of stable somatic gene transfer and the technical challenges of
germline transgenesis. Rapid and stable genetic engineering of the liver would allow
systematic, in vivotesting of contributions by many genes to disease. After fumaryl
acetoacetate hydrolase (Fah) gene transfer to hepatocytes, selective repopulation of the
liver occurs in FAH-deficient mice. This genetic correction is readily mediated with …