Development Of An In Vitro Model Of Hereditary Tyrosinemia Type 1 Using Patient-Derived Induced Pluripotent Stem Cells

Quang Toan Pham,Marie-Agnes M'Callum,Paula Waters,Ugur Halac, Chenicka-Lyn Mangahas,Claudia Raggi,Massimiliano Paganelli

HEPATOLOGY（2017）

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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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