フマリルアセト酢酸ヒドロラーゼ（ＦＡＨ）欠損性ブタおよびその使用

摘要

Described herein is the generation of Fah+/− heterozygote pigs by homologous recombination and somatic cell nuclear transfer, and a method for producing Fah−/− homozygote pigs. The Fah-deficient pigs of the disclosure can be used for a variety of research and therapeutic purposes, such as for the expansion of human hepatocytes, and as large animal models of hereditary tyrosinemia type 1, cirrhosis and hepatocellular carcinoma.