Lentiviral virus-based gene therapy cures hereditary tyrosinemia type 1 in pigs
Oct. 5, 2022
Hereditary tyrosinemia type 1 (HT1) is an inborn error of metabolism caused by deficiency of the fumarylacetoacetate hydrolase (FAH) enzyme and people with it are unable to metabolize the amino acid tyrosine. Toxic metabolites accumulate in the liver leading to severe oxidative damage.