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The combination of Cas9, guide RNA and repair template DNA can induce precise gene editing and the correction of genetic diseases… Expand Preclinical research in animals often fails to adequately predict the outcomes observed in human patients. Chimeric animals… Expand BACKGROUND & AIMS
Hepatocyte-like cells can be derived from pluripotent stem cells such as embryonic stem (ES) cells, but ES cell… Expand The genetic tyrosinemias are characterized by the accumulation of tyrosine in body fluids and tissues. The most severe form of… Expand ABSTRACT Pseudomonas putida metabolizes Phe and Tyr through a peripheral pathway involving hydroxylation of Phe to Tyr (PhhAB… Expand Current strategies for hepatic gene therapy are either quantitatively inefficient or suffer from lack of permanent gene… Expand Hereditary tyrosinaemia type I, a severe autosomal recessive metabolic disease, affects the liver and kidneys and is caused by… Expand BACKGROUND
Hereditary tyrosinemia type I is an autosomal recessive inborn error of metabolism caused by a deficiency of the… Expand Type 1 hereditary tyrosinemia (HT) is an autosomal recessive disease characterized by a deficiency of the enzyme… Expand Succinylacetone (4,6-dioxoheptanoic acid) is an abnormal metabolite produced in patients with hereditary tyrosinemia as a… Expand