ArticleStem cell research2014
Fumarylacetoacetate hydrolase deficient pigs are a novel large animal model of metabolic liver disease.
Article in Stem cell research, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.
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Who cites it
41 citing papers in PubMed, 67 citations in OpenAlex.
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- Transcriptomic Analysis of Liver in Silver sillago,Animals : an open access journal from MDPI · 2023Article
- Patch grafting of organoids of stem/progenitors into solid organs can correct genetic-based disease states.Biomaterials · 2022Article
- In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions.Nature communications · 2022Article
- Advances in pig models of human diseases.Animal models and experimental medicine · 2022Review
- Generation of immunodeficient pig with hereditary tyrosinemia type 1 and their preliminary application for humanized liver.Cell & bioscience · 2022Article
- Limited Expansion of Human Hepatocytes in FAH/Tissue engineering. Part A · 2022Article
- Decreased SLC27A5 Suppresses Lipid Synthesis and Tyrosine Metabolism to Activate the Cell Cycle in Hepatocellular Carcinoma.Biomedicines · 2022Article
- Development ofFrontiers in immunology · 2022Article
- Fumarylacetoacetate hydrolase gene as a knockout target for hepatic chimerism and donor liver production.Stem cell reports · 2021Review
- Survival-Assured Liver Injury Preconditioning (SALIC) Enables Robust Expansion of Human Hepatocytes in FahAdvanced science (Weinheim, Baden-Wurttemberg, Germany) · 2021Article
- Genetically blockingMolecular therapy. Methods & clinical development · 2021Article
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- CRISPR/Cas9-Mediated Gene Correction in Newborn Rabbits with Hereditary Tyrosinemia Type I.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Development of Ectopic Livers by Hepatocyte Transplantation Into Swine Lymph Nodes.Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society · 2020Article
- Article
- Long-Term Outcomes and Practical Considerations in the Pharmacological Management of Tyrosinemia Type 1.Paediatric drugs · 2019Review
- Cold storage of porcine hepatocyte spheroids for spheroid bioartificial liver.Xenotransplantation · 2019Article
Corrections and comments
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Authors and funding
12 authors at 5 institutions in 1 country.
Funding
Abstract
Hereditary tyrosinemia type I (HT1) is caused by deficiency in fumarylacetoacetate hydrolase (FAH), an enzyme that catalyzes the last step of tyrosine metabolism. The most severe form of the disease presents acutely during infancy, and is characterized by severe liver involvement, most commonly resulting in death if untreated. Generation of FAH(+/-) pigs was previously accomplished by adeno-associated virus-mediated gene knockout in fibroblasts and somatic cell nuclear transfer. Subsequently, these animals were outbred and crossed to produce the first FAH(-/-) pigs. FAH-deficiency produced a lethal defect in utero that was corrected by administration of 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3 cyclohexanedione (NTBC) throughout pregnancy. Animals on NTBC were phenotypically normal at birth; however, the animals were euthanized approximately four weeks after withdrawal of NTBC due to clinical decline and physical examination findings of severe liver injury and encephalopathy consistent with acute liver failure. Biochemical and histological analyses, characterized by diffuse and severe hepatocellular damage, confirmed the diagnosis of severe liver injury. FAH(-/-) pigs provide the first genetically engineered large animal model of a metabolic liver disorder. Future applications of FAH(-/-) pigs include discovery research as a large animal model of HT1 and spontaneous acute liver failure, and preclinical testing of the efficacy of liver cell therapies, including transplantation of hepatocytes, liver stem cells, and pluripotent stem cell-derived hepatocytes.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.