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US 11,793,178 B2Granted
Fumarylacetoacetate hydrolase (FAH)-deficient and immunodeficient rats and uses thereof
AssigneeYecuris Corporation
InventorsJohn R. Bial, Elizabeth M. Wilson, Aron M. Geurts
Life of this patent
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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.
Claims14
A method of expanding human hepatocytes in vivo, comprising:
- transplanting human hepatocytes into a first rat, wherein:
- said first rat is 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 first rat,
- and allowing the human hepatocytes to expand in the first rat, thereby expanding human hepatocytes in vivo.
Dependencies are inferred from the claim text.