Hereditary Tyrosinemia Type 1 Mice under Continuous Nitisinone Treatment Display Remnants of an Uncorrected Liver Disease Phenotype

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...

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Bibliographic Details
Main Authors: Jessie Neuckermans, Sien Lequeue, Paul Claes, Anja Heymans, Juliette H. Hughes, Haaike Colemonts-Vroninks, Lionel Marcélis, Georges Casimir, Philippe Goyens, Geert A. Martens, James A. Gallagher, Tamara Vanhaecke, George Bou-Gharios, Joery De Kock
Format: Article
Language:English
Published: MDPI AG 2023-03-01
Series:Genes
Subjects:
liver disease
hereditary tyrosinemia type 1
nitisinone
transcriptomics
alkaptonuria
hepatocellular carcinoma
Online Access:https://www.mdpi.com/2073-4425/14/3/693

Internet

https://www.mdpi.com/2073-4425/14/3/693

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