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Hereditary Tyrosinemia Type 1 Mice under Continuous Nitisinone Treatment Display Remnants of an Uncorrected Liver Disease Phenotype
Hereditary tyrosinemia type 1 (HT1) is a genetic disorder of the tyrosine degradation pathway (TIMD) with unmet therapeutic needs. HT1 patients are unable to fully break down the amino acid tyrosine due to a deficient fumarylacetoacetate hydrolase (FAH) enzyme and, therefore, accumulate toxic tyrosi...
Autores principales: | Neuckermans, Jessie, Lequeue, Sien, Claes, Paul, Heymans, Anja, Hughes, Juliette H., Colemonts-Vroninks, Haaike, Marcélis, Lionel, Casimir, Georges, Goyens, Philippe, Martens, Geert A., Gallagher, James A., Vanhaecke, Tamara, Bou-Gharios, George, De Kock, Joery |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10047938/ https://www.ncbi.nlm.nih.gov/pubmed/36980965 http://dx.doi.org/10.3390/genes14030693 |
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