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...

Descripción completa

Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
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

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10047938/
https://www.ncbi.nlm.nih.gov/pubmed/36980965
http://dx.doi.org/10.3390/genes14030693

Ejemplares similares