ArticleJournal of inherited metabolic disease1998
Therapeutic trials in the murine model of hereditary tyrosinaemia type I: a progress report.
Article in Journal of inherited metabolic disease, 1998. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00865514 (Pharmacotoxicology of Trichloroethylene Metabolites), which is not on this map. Cited by 15 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Pharmacotoxicology of Trichloroethylene Metabolites: Short-term Effect of DCA on in Vivo Tyrosine Catabolism and MAAI Expression
Who cites it
15 citing papers in PubMed, 57 citations in OpenAlex.
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- 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
- Animal Modeling of Pediatric Liver Cancer.Cancers · 2020Review
- The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism.Expert opinion on orphan drugs · 2020Article
- Long-Term Outcomes and Practical Considerations in the Pharmacological Management of Tyrosinemia Type 1.Paediatric drugs · 2019Review
- Senescence and cell death in chronic liver injury: roles and mechanisms underlying hepatocarcinogenesis.Oncotarget · 2018Review
- Diagnosis and treatment of tyrosinemia type I: a US and Canadian consensus group review and recommendations.Genetics in medicine : official journal of the American College of Medical Genetics · 2017Review
- Molecular biology of hepatitis B virus infection.Virology · 2015Review
- Identification of rtl1, a retrotransposon-derived imprinted gene, as a novel driver of hepatocarcinogenesis.PLoS genetics · 2013Article
- Hereditary tyrosinemia type 1 from a single center in Egypt: clinical study of 22 cases.World journal of pediatrics : WJP · 2011Article
- A facile method for somatic, lifelong manipulation of multiple genes in the mouse liver.Hepatology (Baltimore, Md.) · 2008Article
- Liver repopulation and carcinogenesis: two sides of the same coin?The American journal of pathology · 2008Review
- Liver repopulation and correction of metabolic liver disease by transplanted adult mouse pancreatic cells.The American journal of pathology · 2001Article
Corrections and comments
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Authors and funding
4 authors at 2 institutions in 1 country.
Funding
Abstract
We have studied a knockout mouse with fumarylacetoacetate hydrolase (FAH) deficiency as a model of human hereditary tyrosinaemia type (I (HT1). These mice have a phenotype very similar to the human disease, which is characterized by acute hepatic failure, renal tubular disease and hepatocarcinoma. We have previously reported on the efficacy of 2-(2-nitro-4-trifluoromethylbenzyol)-1,3-cyclohexanedione (NTBC) in preventing acute liver disease in HT1 mice. Here we present a progress report on long-term follow up (> 1 year) of high-dose NTBC therapy in combination with tyrosine restriction. In vivo retroviral gene therapy was also effective in abolishing the acute liver failure of HT1. Retrovirally treated mice remained completely healthy and active for 12 months after retroviral gene transfer. However, hepatocarcinoma developed in 2/3 treated animals after 1 year. Southern blot analysis showed that the tumours did not arise from retrovirally transduced hepatocytes but from non-corrected FAH-deficient cells. These results highlight the extreme danger for tumour formation in HT1 and indicate the need for improved gene therapy that leads to the elimination of endogenous FAH-deficient liver cells.
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Registered trials
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