Development Of An In Vitro Model Of Hereditary Tyrosinemia Type 1 Using Patient-Derived Induced Pluripotent Stem Cells
HEPATOLOGY(2017)
Abstract
Background: Hereditary type 1 tyrosinemia (HT1) is a severe inborn error of liver metabolism caused by the deficiency of fumarylacetoacetate hydrolase (FAH). The long-term effects of the only effective treatment available (the herbicide NTBC) are unknown. No in vitro model of HT1 is currently available.
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