Fumarylacetoacetate hydrolase (Fah)-deficient and immunodeficient rats and uses thereof
AssigneeYecuris Corporation
InventorsJohn R. Bial, Elizabeth M. Wilson, Aron M. Geurts
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Abstract
Described herein are rats with a hepatic deficiency comprising decreased function, activity, or expression of an enzyme in the tyrosine catabolic pathway (such as fumarylacetoacetate hydrolase), and methods of using the same for in vivo engraftment and expansion of heterologous hepatocytes, such as human hepatocytes, analysis of human liver disease, and analysis of xenobiotics. Also disclosed is the use of immunodeficient rats for the engraftment and expansion of heterologous hepatocytes.
Claims7
A genetically modified fumarylacetoacetate hydrolase (Fah)-deficient rat whose genome is homozygous for a disruption in exon 3 of the Fah gene such that the disruption results in loss of expression of functional FAH protein and decreased liver function in the rat.
Dependencies are inferred from the claim text.