Immune deficient Fah-knockout mice can be repopulated with human hepatocytes, creating "mice with human livers". These chimeric animals have become an important preclinical model for infectious diseases, metabolism and gene therapy.

Jul 29, 2007 · To provide a broadly useful hepatic xenorepopulation system, we generated severely immunodeficient, fumarylacetoacetate hydrolase (Fah)-deficient mice. After pretreatment with a...

Immunodeficient mice that overexpress urokinase-type plasminogen activator (uPA) and, alternatively, with a knockout of the fumarylacetoacetate hydrolase (Fah) genes are the two most common mouse models for these studies.

In order to address this question we used a mouse model of human tyrosinemia type I with a mutation in the fumarylacetoacetate hydrolase gene (FAH-/-). These mice develop severe liver toxicity and succumb to their metabolic disorder but can be rescued by BMT of wild type (FAH+/+) BM-derived HSC.

Feb 25, 2019 · Intravenous delivery of an adenine base editor and a single-guide RNA for the Fah gene can correct an A>G splice-site mutation in an adult mouse model of tyrosinaemia.

The best available model of liver humanization, the uroplasminogen-activator transgenic model, has major practical limitations. To provide a broadly useful hepatic xenorepopulation system, we generated severely immunodeficient, fumarylacetoacetate hydrolase (Fah)-deficient mice.

To provide a broadly useful hepatic xenorepopulation system, we generated severely immunodeficient, fumarylacetoacetate hydrolase (Fah)-deficient mice. After pretreatment with a urokinase-expressing adenovirus, these animals could be highly engrafted (up to 90%) with human hepatocytes from multiple sources, including liver biopsies.

Explore Cyagen's Fah KO mice for studying tyrosinemia type 1 and liver-related diseases. Ideal for gene therapy and liver stem cell research.

Nov 1, 2018 · This study hypothesized that ex vivo hepatocyte-directed gene editing using CRISPR/Cas9 could be used to correct a mouse model of HT1, in which a single point mutation results in loss of FAH function.

Jul 29, 2017 · Immune deficient Fah-knockout mice can be repopulated with human hepatocytes, creating “mice with human livers”. These chimeric animals have become an important preclinical model for infectious diseases, metabolism and gene therapy.